Publications
| Title | Abstract | Year(sorted ascending) Filter | PMID Filter |
|---|
| functional characterization of a recombinant adeno-associated virus 5-pseudotyped cystic fibrosis transmembrane conductance regulator vector. | despite extensive experience with recombinant adeno-associated virus (raav) 2 vectors in the lung, gene expression has been low in the context of cystic fibrosis (cf) gene therapy, where the large size of the cystic fibrosis transmembrane conductance regulator (cftr) coding sequence has prompted the use of compact endogenous promoter elements. we evaluated the possibility that gene expression from recombinant adeno-associated virus (raav) could be improved by using alternate aav capsid serotypes ... | 2004 | 15353038 |
| augmentation of antitumor activity of a recombinant adeno-associated virus carcinoembryonic antigen vaccine with plasmid adjuvant. | recombinant adeno-associated virus 2 (raav) vectors have been successfully used for sustained expression of therapeutic genes. the potential of using raav as a cancer vaccine vector and the impact of a bacterial plasmid adjuvant on this activity were investigated. c57bl/6 mice received a single intramuscular injection of raav expressing the human tumor-associated antigen, carcinoembryonic antigen (cea). three weeks later, when cea expression was optimal, a bacterial plasmid containing methylated ... | 2004 | 15353040 |
| trans-splicing adeno-associated viral vector-mediated gene therapy is limited by the accumulation of spliced mrna but not by dual vector coinfection efficiency. | therapeutic application of recombinant adeno-associated virus (aav) has been limited by its small carrying capacity. to overcome this limitation trans-splicing vectors were developed recently. however, the transduction efficiency of trans-splicing vectors is considerably lower than that of a single intact vector in skeletal muscle. to improve trans-splicing vectors for skeletal muscle gene therapy, we examined whether coinfection efficiency is a rate-limiting factor in the mdx mouse, a model for ... | 2004 | 15353044 |
| long-term correction of murine lipoprotein lipase deficiency with aav1-mediated gene transfer of the naturally occurring lpl(s447x) beneficial mutation. | human lipoprotein lipase (lpl) deficiency causes profound hypertriglyceridemia and life-threatening pancreatitis. we recently developed an adult murine model for lpl deficiency: lpl -/- mice display grossly elevated plasma triglyceride (tg) levels (>200-fold) and very low high-density lipoprotein cholesterol (hdl-c < 10% of normal). we used this animal model to test the efficacy of adeno-associated virus-mediated expression of hlpl(s447x) (aav1-lpl(s447x)) in muscle for the treatment of lpl defi ... | 2004 | 15353045 |
| osteoarthritis gene therapy. | osteoarthritis (oa) is the western world's leading cause of disability. it is incurable, costly and responds poorly to treatment. this review discusses strategies for treating oa by gene therapy. as oa affects a limited number of weight-bearing joints and has no major extra-articular manifestations, it is well suited to local, intra-articular gene therapy. possible intra-articular sites of gene transfer include the synovium and the cartilage. most experimental progress has been made with gene tr ... | 2004 | 14724685 |
| residues within the b' motif are critical for dna binding by the superfamily 3 helicase rep40 of adeno-associated virus type 2. | we have recently published the crystal structure of the adeno-associated virus type 2 superfamily 3 (sf3) helicase rep40. although based on its biochemical properties it is unlikely that rep40 plays a central role as a replicative helicase the involvement of this motor protein in dna packaging has recently been demonstrated. here we focused our attention on residues that fall within and adjacent to the b' motif of sf3 helicases that directly interact with single-stranded dna during translocation ... | 2004 | 15371437 |
| feasibility of gene therapy in gaucher disease using an adeno-associated virus vector. | gaucher disease, one of the common lysosomal storage disorders, is caused by a deficiency of glucocerebrosidase (gc). we investigated gene transfer using recombinant adeno-associated viral (raav) vectors containing human gc cdna driven by the human elongation factor 1-alpha promoter. this raav vector mediated efficient expression of human gc in human gaucher fibroblasts. gc activities were increased from 2.8 to 3.4 times in normal fibroblast and from 1.9 to 4.6 times in gaucher fibroblasts, and ... | 2004 | 15372321 |
| analysis of the production efficiency and titration of various recombinant adeno-associated viruses. | recombinant adeno-associated virus type 2 (raav2) viral vector, a non-pathogenic human parvovirus, has recently emerged as a gene transfer vehicle for cancer gene therapy. to utilize raav2 properly and safely while carrying out preclinical and clinical studies, it is crucial to exactly titer the virus. we therefore compared biological infectious raav2 titers with physical titers of raav2 vectors encoding various transgenes with different sized viral genomes. biological raav2 infectivity was assa ... | 2004 | 15375497 |
| purification of recombinant adeno-associated virus type 8 vectors by ion exchange chromatography generates clinical grade vector stock. | recombinant vectors based on the recently isolated aav serotype 8 (raav-8) shows great promise for gene therapy, particularly for disorders affecting the liver. transition of this vector system to the clinic, however, is limited by the lack of an efficient scaleable purification method. in this report, we describe a simple method for purification of raav-8 vector particles based on ion exchange chromatography that generates vector stocks with greater than 90% purity. the average yield of purifie ... | 2004 | 15381358 |
| progress in the use of adeno-associated viral vectors for gene therapy. | the development of safe and efficient gene transfer vectors is crucial for the success of gene therapy trials. a viral vector system promising to meet these requirements is based on the apathogenic adeno-associated virus (aav-2), a member of the parvovirus family. the advantages of this vector system is the stability of the viral capsid, the low immunogenicity, the ability to transduce both dividing and non-dividing cells, the potential to integrate site specifically and to achieve long-term gen ... | 2004 | 15388988 |
| recombinant adeno-associated virus 2-mediated transfer of the human superoxide-dismutase gene does not confer radioresistance on hela cervical carcinoma cells. | the success rate of any therapeutic approach depends on the therapeutic window, which can be increased by either raising the resistance of the normal tissue without protecting the tumor cells or by sensitizing the tumor cells but not the normal cells. two promising candidate genes for normal tissue protection against radiation-induced damage may be the copper-zinc (cuznsod) and manganese superoxide-dismutase genes (mnsod). the recombinant adeno-associated virus 2 (raav-2) offers attractive advan ... | 2004 | 15450734 |
| site-specific integration of functional transgenes into the human genome by adeno/aav hybrid vectors. | uncontrolled insertion of gene transfer vectors into the human genome is raising significant safety concerns for their clinical use. the wild-type adeno-associated virus (aav) can insert its genome at a specific site in human chromosome 19 (aavs1) through the activity of a specific replicase/integrase protein (rep) binding both the aavs1 and the viral inverted terminal repeats (itrs). aav-derived vectors, however, do not carry the rep gene and cannot maintain site-specific integration properties ... | 2004 | 15451450 |
| efficient transduction of skeletal muscle using vectors based on adeno-associated virus serotype 6. | vectors based on recombinant adeno-associated viruses (raav) have emerged as tools of choice for gene transfer to skeletal muscle. raav vectors demonstrate efficient, safe, and stable transduction. multiple serotypes of aav exist, but vectors based on serotype 2 (raav2) are the most thoroughly characterized and frequently employed. here, we characterize transduction of the skeletal musculature using raav vectors pseudotyped with serotype 6 capsid proteins (raav6). we demonstrate that raav6 vecto ... | 2004 | 15451451 |
| photoreceptor protection by iris pigment epithelial transplantation transduced with aav-mediated brain-derived neurotrophic factor gene. | to determine whether subretinal transplantation of iris pigment epithelial (ipe) cells transduced with the adeno-associated virus (aav2)-mediated brain-derived neurotrophic factor (bdnf) gene can protect photoreceptors against phototoxicity. | 2004 | 15452082 |
| splice junction map of simian parvovirus transcripts. | the transcription map of simian parvovirus (spv), an erythrovirus similar to parvovirus b19, was investigated. rna was extracted from tissues of experimentally infected cynomolgus macaques and subjected to reverse transcription-pcr with spv-specific primers. the pcr products were cloned and sequenced to identify splice junctions. a total of 14 distinct sequences were identified as putative partial transcripts. of these, 13 were spliced; a single unspliced transcript putatively encoded ns1. seque ... | 2004 | 15452211 |
| herpes simplex virus type 1 icp0 protein mediates activation of adeno-associated virus type 2 rep gene expression from a latent integrated form. | adeno-associated virus type 2 (aav-2) is a human parvovirus that requires the presence of a helper virus, such as the herpes simplex virus type 1 (hsv-1) to accomplish a complete productive cycle. in the absence of helper virus, aav-2 can establish a latent infection that is characterized by the absence of expression of viral genes. so far, four hsv-1 early genes, ul5/8/52 (helicase primase complex) and ul29 (single-stranded dna-binding protein), were defined as sufficient for aav replication wh ... | 2004 | 15452218 |
| recombinant aav-mediated gene transfer to the retina: gene therapy perspectives. | retinal degenerative diseases such as retinal macular degeneration and retinitis pigmentosa constitute a broad group of diseases that all share one critical feature, the progressive apoptotic loss of cells in the retina. there is currently no effective treatment available by which the course of these disorders can be modified, and visual dysfunction often progresses to total blindness. gene therapy represents an attractive approach to treating retinal degeneration because the eye is easily acces ... | 2004 | 15454954 |
| isolation of an adenovirus and an adeno-associated virus from goat kids with enteritis. | a dairy goat operation in minnesota experienced a sudden, markedly increased mortality among its neonatal goats. approximately 60 of 130 kids (46%) died. the animals had diarrhea and dyspnea of 1-2 days duration before death. necropsy of 4 goat kids revealed marked, acute, catarrhal enteritis and fibrinous pleuropneumonia. mannheimia haemolytica was isolated from the lungs. basophilic inclusion bodies filling the entire nucleus were present in enterocytes of the ileum of 3 goats. adenoviral part ... | 2004 | 15460334 |
| large-scale production, purification and crystallization of wild-type adeno-associated virus-2. | adeno-associated virus-2 (aav-2) has long been recognized as a potential vector for human gene therapy. although much progress has been made in the molecular virology of aav-2, structural studies of aav-2 have been hampered by the low efficiency of virus production in culture, the low purity of preparations, and the low solubility of pure virus particles in solution. methods of larger scale aav-2 production have been developed through adaptation to suspension culture and re-optimization of the t ... | 2004 | 15488616 |
| non-viral vectors in cystic fibrosis gene therapy: progress and challenges. | although the viability of cystic fibrosis (cf) gene transfer to airway epithelium has been demonstrated in vitro and in animal models, so far none of the clinical investigations using adenovirus, adeno-associated virus, lentivirus, cationic lipids or polymers has shown a persistent correction of the ion transport defects that occur in cf. despite disappointing results, these studies have shown that non-viral vectors could represent a viable alternative for gene therapy in cf airway epithelium. t ... | 2004 | 15491803 |
| adeno-associated viral vector-mediated expression of endostatin inhibits tumor growth and metastasis in an orthotropic pancreatic cancer model in hamsters. | we examined the feasibility of using adeno-associated virus (aav)-mediated systemic delivery of endostatin in gene therapy to treat metastasis of pancreatic cancer. we established an animal model of orthotopic metastatic pancreatic cancer in which the pancreatic cancer cell line pgham-1 was inoculated into the pancreas of syrian golden hamsters. transplanted cells proliferated rapidly and metastasized to the liver. an aav vector expressing endostatin (5 x 10(10) particles) was injected intramusc ... | 2004 | 15492274 |
| adeno-associated virus vector-mediated interleukin-10 gene transfer inhibits atherosclerosis in apolipoprotein e-deficient mice. | inflammation is a major contributor to atherosclerosis by its effects on arterial wall biology and lipoprotein metabolism. interleukin-10 (il-10) is an anti-inflammatory cytokine that may modulate the atherosclerotic disease process. we investigated the effects of adeno-associated virus (aav) vector-mediated gene transfer of il-10 on atherogenesis in apolipoprotein e (apoe)-deficient mice. a murine myoblast cell line, c2c12, transduced with aav encoding murine il-10 (aav2-mil10) secreted substan ... | 2004 | 15496963 |
| [gene therapy of tyrosine hydroxylase, aromatic l-amino acid decarboxylase, and gtp cyclohydrolase genes in rat model of parkinson's disease]. | to detect the expression and function of enzyme genes involved in biosynthetic pathway for dopamine in vitro and assess their effect in rat model of parkinson's disease. | 2004 | 15500713 |
| development of a safe oral abeta vaccine using recombinant adeno-associated virus vector for alzheimer's disease. | a new oral vaccine for alzheimer's disease was developed using recombinant adeno-associated virus vector carrying abeta cdna (aav/abeta). oral administration of the vaccine without adjuvant induced the expression and secretion of abeta1-43 or abeta1-21 in the epithelial cell layer of the intestine in amyloid precursor protein transgenic mice. serum antibody levels were elevated for more than six months, while t cell proliferative responses to abeta was not detected. brain abeta burden was signif ... | 2004 | 15505369 |
| evaluation of exposure and health care worker response to nebulized administration of tgaavcf to patients with cystic fibrosis. | a study was conducted to assess health care worker exposure to tgaavcf during the aerosolized administration of this experimental gene transfer agent in clinical trials for the treatment of cystic fibrosis (cf). tgaavcf is a recombinant adeno-associated virus (aav) genetically engineered to contain the human cf transmembrane conductance regulator cdna. study subjects included eight health care workers involved in the administration of tgaavcf in a phase ii study and 12 control health care worker ... | 2004 | 15507460 |
| secretion of a tnfr:fc fusion protein following pulmonary administration of pseudotyped adeno-associated virus vectors. | this study evaluated and compared delivery of the tumor necrosis factor alpha receptor (tnfr)-immunoglobulin g1 (igg1) fc fusion (tnfr:fc) gene to the lung by single and repeat administrations of multiple pseudotyped adeno-associated virus (aav) vectors as a means for achieving systemic distribution of the soluble tnfr:fc protein. a single endotracheal administration of aav[2/5]cytomegalovirus (cmv)-tnfr:fc vector (containing the aav2 inverted terminal repeats and aav5 capsid) to the rat lung re ... | 2004 | 15507622 |
| aav vector-mediated microdystrophin expression in a relatively small percentage of mdx myofibers improved the mdx phenotype. | duchenne muscular dystrophy (dmd) is a lethal disorder of skeletal muscle caused by mutations in the dystrophin gene. adeno-associated virus (aav) vector-mediated gene therapy is a promising approach to the disease. although a rod-truncated microdystrophin gene has been proven to ameliorate dystrophic phenotypes, the level of microdystrophin expression required for effective gene therapy by an aav vector has not been determined yet. here, we constructed a recombinant aav type 2 vector, aav2-mckd ... | 2004 | 15509500 |
| vascular endothelial growth factor stimulates skeletal muscle regeneration in vivo. | vascular endothelial growth factor (vegf) is a major regulator of blood vessel formation during development and in the adult organism. recent evidence indicates that this factor also plays an important role in sustaining the proliferation and differentiation of different cell types, including progenitor cells of different tissues, including bone marrow, bone, and the central nervous system. here we show that the delivery of the 165-aa isoform of vegf-a cdna using an adeno-associated virus (aav) ... | 2004 | 15509502 |
| self-complementary adeno-associated virus 2 (aav)-t cell protein tyrosine phosphatase vectors as helper viruses to improve transduction efficiency of conventional single-stranded aav vectors in vitro and in vivo. | recombinant vectors based on adeno-associated virus type 2 (aav) target the liver efficiently, but the transgene expression is limited to approximately 5% of hepatocytes. the lack of efficient transduction is due, in part, to the presence of a cellular protein, fkbp52, phosphorylated forms of which inhibit the viral second-strand dna synthesis. we have documented that dephosphorylation of fkbp52 at tyrosine residues by the cellular t cell protein tyrosine phosphatase (tc-ptp) enhances aav-mediat ... | 2004 | 15509512 |
| advances in cystic fibrosis gene therapy. | the first cystic fibrosis gene therapy trials were carried out in 1993, and although proof-of-principle for gene transfer to the lungs was established, efficiency was generally low. the authors review the most recent advances in preclinical airway gene transfer and summarize the results from the latest clinical trials. | 2004 | 15510064 |
| recombinant adeno-associated virus serotype 2 effectively transduces primary rat brain astrocytes and microglia. | recombinant adeno-associated virus-2 (raav2) under control of the chicken beta actin promoter/truncated cmv enhancer (cba) was investigated for its ability to transduce primary cultures of rat brain neurons, microglia and astrocytes. this vector was highly effective in all three cell types in heparin-sensitive manners (astrocytes, microglia and neurons transduced by >98%, 75%, and 95%, respectively). however, astrocytes co-cultured with neurons were not transduced. raav2/cba is an important new ... | 2004 | 15519948 |
| inhibition of human immunodeficiency virus type 1 replication by sirna targeted to the highly conserved primer binding site. | the initiation of hiv-1 reverse transcription occurs at an 18-nucleotide sequence in the viral genome designated as the primer binding site (pbs), which is complementary to the 3' terminal nucleotides of trna(lys,3). since the pbs is highly conserved among all infectious hiv-1, it represents an attractive target for the development of new therapeutics to inhibit viral replication. in this study, we have evaluated three approaches using small interfering rna (sirnas) targeted to the pbs for the c ... | 2004 | 15527848 |
| sumo-1 modification regulates the protein stability of the large regulatory protein rep78 of adeno associated virus type 2 (aav-2). | the large rep proteins rep78 and rep68 of the helper-dependent adeno associated virus type 2 (aav-2) are essential for both site-specific integration of aav dna in the absence of helpervirus and productive aav replication in the presence of helpervirus. we have identified ubc9, the e2 conjugating enzyme for the small ubiquitin-related polypeptide sumo-1, as binding partner of the large rep proteins in yeast two-hybrid analysis and in gst pulldown assays. modification of the large rep proteins wi ... | 2004 | 15527853 |
| a novel approach to quantitative ultrasonic naked gene delivery and its non-invasive assessment. | the purpose of this study was to investigate practical, safe, easy-to-use, non-cytotoxic, and reliable parameters to apply to an ultrasound (us) naked gene therapy system. the ultrasound pressure at the point of cell exposure was measured using a calibrated hydrophone and the intensity calculated. an acoustic power meter calibrated using a hydrophone was used to measure the power of the transducer. four cell types were exposed to us with different exposure times and intensities. fluorescent micr ... | 2004 | 15530980 |
| adeno-associated viral vector delivers cardiac-specific and hypoxia-inducible vegf expression in ischemic mouse hearts. | it has been shown that the adeno-associated virus (aav) vector can deliver the vegf gene efficiently into the ischemic mouse myocardium. however, the aav genomes can be found in extracardiac organs after intramyocardial injection. to limit unwanted vegf expression in organs other than the heart, we tested the use of the cardiac myosin light chain 2v (mlc-2v) promoter and the hypoxia-response element to mediate cardiac-specific and hypoxia-inducible vegf expression. an aav vector, mlcvegf, with 2 ... | 2004 | 15534198 |
| gene therapy for optic nerve disease. | there has been recent interest in the potential use of gene therapy techniques to treat ocular disease. in this article, we consider the optic nerve diseases that are potentially most amenable to gene therapy. | 2004 | 15534589 |
| improved behavior and neuropathology in the mouse model of sanfilippo type iiib disease after adeno-associated virus-mediated gene transfer in the striatum. | sanfilippo syndrome is a mucopolysaccharidosis (mps) caused by a lysosomal enzyme defect interrupting the degradation pathway of heparan sulfates. affected children develop hyperactivity, aggressiveness, delayed development, and severe neuropathology. we observed relevant behaviors in the mouse model of sanfilippo syndrome type b (mpsiiib), in which the gene coding for alpha-n-acetylglucosaminidase (naglu) is invalidated. we addressed the feasibility of gene therapy in these animals. vectors der ... | 2004 | 15537895 |
| development and characterization of a cell line for large-scale, serum-free production of recombinant adeno-associated viral vectors. | one of the major limitations to the use of adeno-associated virus (aav) vectors for gene therapy has been the difficulty in producing enough vector to supply a clinical trial. more than 20 000 roller bottles may be required to generate aav by the traditional transient transfection process to treat 50 patients. a scalable aav producer cell line grown in serum-free media will meet the needs for the manufacture of aav gene therapeutics. | 2004 | 15538729 |
| bioeffects of low-frequency ultrasonic gene delivery and safety on cell membrane permeability control. | to develop a novel method of ultrasonic naked gene delivery (ungd); to examine the relationship between optimal parameters of ultrasound exposure and cell membrane permeability, enzymes, and free radicals; and to find optimal control parameters that were realizable, reliable, and noncytotoxic for use in gene therapy. | 2004 | 15557300 |
| recent advance in hepatic insulin gene therapy. | the development of type 1 diabetes results from the almost total destruction of insulin-producing pancreatic cells by autoimmune responses specific to cells. standard insulin therapy may not maintain blood glucose concentrations within the relatively narrow range that occurs in the presence of normal pancreatic cells. we used a recombinant adeno-associated virus (raav) that expresses a single-chain insulin analogue (sia), which possesses biologically active insulin activity without enzymatic con ... | 2004 | 15563976 |
| adeno-associated virus and the development of adeno-associated virus vectors: a historical perspective. | 2004 | 15564130 | |
| dual therapeutic utility of proteasome modulating agents for pharmaco-gene therapy of the cystic fibrosis airway. | pharmacologic- and gene-based therapies have historically been developed as two independent therapeutic platforms for cystic fibrosis (cf) lung disease. inhibition of the dysregulated epithelial na channel (enac) is one pharmacologic approach to enhance airway clearance in cf. we investigated pharmacologic approaches to enhance cftr gene delivery with recombinant adeno-associated virus (raav) and identified compounds that significantly improved viral transduction while simultaneously inhibiting ... | 2004 | 15564131 |
| intrapleural administration of a serotype 5 adeno-associated virus coding for alpha1-antitrypsin mediates persistent, high lung and serum levels of alpha1-antitrypsin. | alpha1-antitrypsin (alpha1at) is a serine proteinase inhibitor that protects the lung from degradation by neutrophil proteases. in alpha1at deficiency, an autosomal recessive disorder resulting from mutations in the alpha1at (approved symbol serpina1) gene, serum alpha1at levels of < 570 microg/ml are associated with development of emphysema. adeno-associated virus (aav) serotype 2 (aav2) vectors expressing alpha1at administered intramuscularly or intravenously mediate sustained serum levels of ... | 2004 | 15564132 |
| intracellular viral processing, not single-stranded dna accumulation, is crucial for recombinant adeno-associated virus transduction. | adeno-associated virus (aav) is a unique gene transfer vector which takes approximately 4 to 6 weeks to reach its expression plateau. the mechanism for this slow-rise expression profile was proposed to be inefficient second-strand dna synthesis from the input single-stranded (ss) dna viral genome. in order to clarify the status of ss aav genomes, we generated aav vectors labeled with bromodeoxyuridine (brdu), a nucleotide analog that can be incorporated into the aav genome and packaged into infe ... | 2004 | 15564477 |
| efficient early and sustained transduction of human fetal mesencephalon using adeno-associated virus type 2 vectors. | the success of transplantation of human fetal mesencephalic tissue into the putamen of patients with parkinson's disease (pd) is still limited by the poor survival of the graft. in animal models of fetal transplantation for pd, antiapoptotic agents, such as growth factors or caspase inhibitors, or agents counteracting oxidative stress enhance the survival and reinnervation potential of the graft. genetic modification of the transplant could allow a local and continuous delivery of these factors ... | 2004 | 15565868 |
| integration of adeno-associated virus (aav) and recombinant aav vectors. | the driving interest in adeno-associated virus (aav) has been its potential as a gene delivery vector. the early observation that aav can establish a latent infection by integrating into the host chromosome has been central to this interest. however, chromosomal integration is a two-edged sword, imparting on one hand the ability to maintain the therapeutic gene in progeny cells, and on the other hand, the risk of mutations that are deleterious to the host. a clearer understanding of the mechanis ... | 2004 | 15568995 |
| [expression of trail(114-281) mediated by adeno-associated virus and its tumoricidal activity]. | to investigate the expression of the soluble tumor necrosis factor-related apoptosis-inducing ligand (trail) mediated by adeno-associated virus (aav) and its tumoricidal activity in vitro and vivo. | 2004 | 15569460 |
| menisci are efficiently transduced by recombinant adeno-associated virus vectors in vitro and in vivo. | meniscal tears remain an unsolved problem in sports medicine. gene transfer is a potential approach to enhancing meniscal repair. recombinant adeno-associated virus is a method of gene transfer that has advantages over previously used approaches to this problem. | 2004 | 15572313 |
| herpes simplex virus type 1 amplicons and their hybrid virus partners, ebv, aav, and retrovirus. | hsv-1 amplicons can accommodate foreign dna of any size up to 150 kbp. genomic sequences as well as cdna, large transcriptional regulatory sequences for cell type-specific expression, or multiple transgenes can be inserted in a modular fashion. hsv-1 amplicon vectors deliver dna efficiently into the cell nucleus as an extrachromosomal, non-replicating circular concatenate, which is rapidly diluted, at least in dividing cells. consequently, transgene expression is lost within days to weeks in div ... | 2004 | 15578989 |
| [study on the recombinant adeno-associated virus vector carrying lacz gene expression in the skeletal muscle]. | to look for a gene delivery route to the treatment of duchenne muscular dystrophy(dmd). | 2004 | 15583992 |
| sonic hedgehog delivered by an adeno-associated virus protects dopaminergic neurones against 6-ohda toxicity in the rat. | direct intracerebral administration of sonic hedgehog (shh) reduces 6-ohda and mptp toxicity to nigral dopaminergic cells in rats and primates. to determine whether transfection of the dna sequence for shh using viral vectors also protects against 6-ohda toxicity, a type 2 adeno- associated virus (aav) incorporating 600 base pairs of n-terminal shh dna was generated to induce shh expression in rat striatum.aav-shh was injected into the striatum, 3 weeks prior to the initiation of an unilateral p ... | 2004 | 15586304 |
| clinical protocol. administration of a replication-deficient adeno-associated virus gene transfer vector expressing the human cln2 cdna to the brain of children with late infantile neuronal ceroid lipofuscinosis. | late infantile neuronal ceroid lipofuscinosis (lincl) is a fatal childhood neurodegenerative lysosomal storage disease with no known therapy. there are estimated to be 200 to 300 children in the united states at any one time with the disease. lincl is a genetic disease resulting from a deficiency of tripeptidyl peptidase i (tpp-i), a proteolytic enzyme encoded by cln2, the gene that is mutated in individuals with lincl. the subjects are chronically ill, with a progressive cns disorder that invar ... | 2004 | 15610613 |
| [immune potency of recombinant adeno-associated virus combined with recombinant adenovirus vaccine containing hiv-1 gp120]. | to study the immune effect of recombinant adeno-associated virus (raav) combined with recombinant adenovirus (radv) vaccine in balb/c mice. | 2004 | 15650777 |
| [treatment of alzheimer disease: a beta vaccine]. | alzheimer disease (ad) is regarded as an amyloidosis of the brain, and, therefore, the prevention/deletion of beta amyloid deposition is one of the most promising target of the treatment. particularly, immune-mediated strategy is now known as a beta vaccination, which is thought to be a highly feasible way being applicable to ad patients. the a beta vaccination was originated by schenk et al. in 1999. since active and passive immunization of amyloid precursor protein (app) gene- transgenic mice ... | 2004 | 15651289 |
| [current status and perspective of gene therapy on dystrophic animal model]. | duchenne muscular dystrophy (dmd) is an x-linked, lethal muscle disorder caused by mutations in the dystrophin gene. an adeno-associated virus (aav) vector-mediated gene transfer is one of attractive approaches to the treatment of dmd, though it has a limitation in insertion size up to 4.9 kb. therefore, a full-length dystrophin cdna (14 kb) cannot be incorporated into an aav vector. we previously generated micro-dystrophin transgenic dystrophin-deficient mdx mice. micro-dystrophin cs1 transgeni ... | 2004 | 15651329 |
| [progress in pathogenesis and therapeutic research in retinitis pigmentosa and age-related macular degeneration]. | retinitis pigmentosa (rp) and age-related macular degeneration (amd) are designated special targeted eye diseases by the welfare and labor ministry of japan. we have been studying the pathogenesis, diagnosis, treatment, and evaluation of these diseases. the development of molecular genetic analyses of rp revealed that the type and frequency of mutations varied with the ethnic population. in our present study, we focused on the genetic analysis and clinical examinations for autosomal dominant ret ... | 2004 | 15656086 |
| generation of recombinant skin in vitro by adeno-associated virus type 2 vector transduction. | it has long been recognized that skin may be a particularly good target for pharmacologic gene therapy and as a platform for the secretion of systemically distributed molecules. adeno-associated virus type 2 (aav) is a useful vector for skin gene therapy because skin is the natural host tissue for aav, in which it functions as an autonomous parvovirus. we demonstrate here that recombinant (r) aav vectors carrying the granulocyte-macrophage colony-stimulating factor (gm-csf), human papillomavirus ... | 2004 | 15684679 |
| osteogenic differentiation of recombinant adeno-associated virus 2-transduced murine mesenchymal stem cells and development of an immunocompetent mouse model for ex vivo osteoporosis gene therapy. | gene therapy for osteopenic conditions including osteoporosis is a potential alternative to pharmacotherapy for cost effectiveness, long-term viability, and the ability to enhance bone mass by anabolic approaches. increased understanding of mesenchymal stem cell (msc) lineage differentiation during osteogenesis, and of the molecular pathways involved in bone cell production, provides an opportunity for the advancement of gene therapy approaches for osteopenic conditions. the potential of mscs in ... | 2004 | 15684696 |
| impaired nuclear transport and uncoating limit recombinant adeno-associated virus 2 vector-mediated transduction of primary murine hematopoietic cells. | controversies abound concerning hematopoietic stem cell transduction by recombinant adeno-associated virus 2 (aav) vectors. for human hematopoietic cells, we have shown that this problem is related to the extent of expression of the cellular receptor for aav. at least a small subset of murine hematopoietic cells, on the other hand, does express both the aav receptor and the coreceptor, yet is transduced poorly. in the present study, we have found that approximately 85% of aav genomes were presen ... | 2004 | 15684697 |
| effects of raav-cd151 and raav-anticd151 on the migration of human tongue squamous carcinoma cell line tca8113. | this study was designed to determine the effects of the recombinant adeno-associated virus vector containing sense cd151 gene (raav-cd151) and antisense cd151 gene (raav-anticd151) on the migration of tca8113 cell. functional fragment of cd151 gene was amplified by rt-pcr, and inserted into the vector paav in the sense direction and antisense direction, respectively. the raav-cd151 and raav-anticd151 were produced and the titers were determined by dot blot. the cd151, at protein level, was detec ... | 2004 | 15791839 |
| gene therapy in epilepsy. | the generation of viral vectors, such as adeno-associated virus (aav) and lentivirus, which are capable of stable transduction of neurons, offers an attractive strategy for introducing novel genes into the brain, resulting in a long-lasting production of specific proteins. an alternative approach to achieving transgene expression in brain is to graft cells that are genetically engineered to produce neuroactive substances. neuroactive peptides, adenosine, and gamma-aminobutyric acid, are agents t ... | 2004 | 16059458 |
| [immune response induced by hiv dna vaccine combined with recombinant adeno-associated virus]. | hiv-1 dna vaccine and recombinant adeno-associated virus (raav) expressing gagv3 gene of hiv-1 subtype b were constructed and balb/c mice were immunized by vaccination regimen consisting of consecutive priming with dna vaccine and boosting with raav vaccine; the ctl and antibody response were detected and compared with those induced by dna vaccine or raav vaccine separately. | 2004 | 15640860 |
| adeno-associated virus and lentivirus pseudotypes for lung-directed gene therapy. | the enthusiasm for cystic fibrosis gene therapy that attended the initial cloning of the gene and in vitro correction of the genetic defect eventually diminished as we learned more about the limitations of vector technologies that were available in the 1980s and 1990s. substantial progress has been made, however, over the last 5 years in developing second- and third-generation vector constructs that should be more useful in achieving gene transfer to the lung for the treatment of pulmonary disea ... | 2004 | 16113451 |
| susceptibility of mesothelioma cell lines to adeno-associated virus 2 vector-based suicide gene therapy. | although great efforts have been made to improve conventional therapy for diffuse malignant pleural mesothelioma, the median survival time of the patients after appearance of clinical symptoms remains poor. due to confinement of the primary tumor to the pleural space, locoregional approaches are attractive strategies to improve the clinical outcome. in this context locoregional gene therapy using the recombinant adeno-associated virus 2 (raav-2) may be a new approach. vectors were constructed co ... | 2004 | 15474666 |
| recombinant adeno-associated virus preferentially transduces human, compared to mouse, synovium: implications for arthritis therapy. | despite a number of published reports, including from our own laboratory, suggesting that adeno-associated virus (aav) transduces mouse synovium, a careful analysis demonstrated transduction predominantly of the subsynovial muscle tissue, while the synovial lining is poorly transduced. to investigate the potential of aav to transduce human synovium, three human rheumatoid arthritis (ra) and two murine collagen-induced arthritis (cia) synovial cell lines were infected with recombinant aav (raav) ... | 2004 | 17028800 |
| [the experimental study of raav2 transfecting neural stem cells-drived neurospheres]. | to investigate the abilities of recombinant adeno-associated virus type 2 (raav2) transfecting neurospheres. | 2004 | 21166205 |
| molecular imaging and contrast agent database (micad) | a potential approach to the treatment of diseases and genetic disorders in humans is gene therapy [pubmed]. this is a technique whereby the absent or malfunctioned gene is replaced by a working gene to produce an enzyme or protein to correct the progression of the disease. noninvasive molecular imaging technologies play an important role in the fields of gene therapy by monitoring gene expression continuously in living animals (1). magnetic resonance imaging (mri), optical imaging, ultrasound im ... | 2004 | 20641413 |
| molecular imaging and contrast agent database (micad) | a potential approach to the treatment of diseases and genetic disorders in humans is gene therapy [pubmed]. this is a technique whereby the absent or malfunctioning gene is replaced by a working gene to produce an enzyme or a protein to correct the progression of disease. noninvasive molecular imaging technologies play an important role in the fields of gene therapy by monitoring gene expression continuously in living animals (1). magnetic resonance imaging (mri), optical imaging, ultrasound ima ... | 2004 | 20641753 |
| the hypothalamic arcuate nucleus: a key site for mediating leptin's effects on glucose homeostasis and locomotor activity. | leptin is required for normal energy and glucose homeostasis. the hypothalamic arcuate nucleus (arh) has been proposed as an important site of leptin action. to assess the physiological significance of leptin signaling in the arh, we used mice homozygous for a flpe-reactivatable, leptin receptor null allele (lepr(neo/neo) mice). similar to lepr(db/db) mice, these mice are obese, hyperglycemic, hyperinsulinemic, infertile, and hypoactive. to selectively restore leptin signaling in the arh, we gen ... | 2005 | 16054045 |
| production, purification, crystallization and preliminary x-ray analysis of adeno-associated virus serotype 8. | adeno-associated viruses (aavs) are actively being developed for clinical gene-therapy applications and the efficiencies of the vectors could be significantly improved by a detailed understanding of their viral capsid structures and the structural determinants of their tissue-transduction interactions. aav8 is approximately 80% identical to the more widely studied aav2, but its liver-transduction efficiency is significantly greater than that of aav2 and other serotypes. the production, purificat ... | 2005 | 16511095 |
| production, purification, crystallization and preliminary x-ray structural studies of adeno-associated virus serotype 5. | adeno-associated virus serotype 5 (aav5) is under development for gene-therapy applications for the treatment of cystic fibrosis. to elucidate the structural features of aav5 that control its enhanced transduction of the apical surface of airway epithelia compared with other aav serotypes, x-ray crystallographic studies of the viral capsid have been initiated. the production, purification, crystallization and preliminary crystallographic analysis of empty aav5 viral capsids are reported. the cry ... | 2005 | 16511195 |
| adeno-associated virus as a gene therapy vector: vector development, production and clinical applications. | adeno-associated virus (aav) has emerged as an attractive vector for gene therapy. aav vectors have successfully been utilized to promote sustained gene expression in a variety of tissues such as muscle, eye, brain, liver, and lung. as the significance of aav as a gene therapy vector has been realized over the past years, recent developments in recombinant aav (raav) production and purification have revolutionized the aav field. it is now possible to produce high yields of vector (10(12)-10(13) ... | 2005 | 16568890 |
| advanced targeting strategies for murine retroviral and adeno-associated viral vectors. | targeted gene delivery involves broadening viral tropism to infect previously nonpermissive cells, replacing viral tropism to infect a target cell exclusively, or stealthing the vector against nonspecific interactions with host cells and proteins. these approaches offer the potential advantages of enhanced therapeutic effects, reduced side effects, lowered dosages, and enhanced therapeutic economics. this review will discuss a variety of targeting strategies, both genetic and nongenetic, for re- ... | 2005 | 16568891 |
| [adeno-associated virus-mediated gene transfer of endostatin for inhibiting growth and metastasis of human nasopharyngeal carcinoma in nude mice]. | to investigate the inhibitory effect on growth and metastasis of human nasopharyngeal carcinoma in nude mice by adeno-associated virus-mediated gene transfer of human endostatin. | 2005 | 16874953 |
| precise hit: adeno-associated virus in gene targeting. | vectors based on the adeno-associated virus (aav) have attracted much attention as potent gene-delivery vehicles, mainly because of the persistence of this non-pathogenic virus in the host cell and its sustainable therapeutic gene expression. however, virus infection can be accompanied by potentially mutagenic random vector integration into the genome. a novel approach to aav-mediated gene therapy based on gene targeting through homologous recombination allows efficient, high-fidelity, non-mutag ... | 2005 | 16261169 |
| identification of mouse aav capsid-specific cd8+ t cell epitopes. | adeno-associated virus has been developed for use as a gene transfer vector. to understand the impact of aav capsid-specific cd8(+) t cells on aav-mediated gene transfer, we identified cd8(+) t cell epitopes for aav-2 and aav-8 capsid in c57bl/6 (h-2(b) mhc haplotype) and balb/c (h-2(d) mhc haplotype) mice. mice of both the h-2(b) and the h-2(d) haplotypes recognized epitopes on aav-2 and aav-8 capsid. t cells from h-2(b) mice recognized an epitope that was conserved between aav-2 and aav-8 caps ... | 2005 | 16263332 |
| treatment with hydroxyurea and tyrphostin-1 significantly improves the transduction efficiency of recombinant adeno-associated viruses in human cancer cells. | to enhance the transduction efficiency (te) of a recombinant adeno-associated virus 2 (raav2) in human cancer cells, we examined the combined effects of various chemicals known to influence the raav2 transduction process at distinct steps. among the agents tested were trichostatin a, a histone deacetylase inhibitor, mg-132, a proteosome inhibitor, the genotoxic agents hydroxyurea, aphidicolin, etoposide and camptothecin, and tyrphostin-1, an epidermal growth factor receptor inhibitor. during or ... | 2005 | 16273241 |
| [effect of secretory human calcitonin gene-related peptide recombinant aav on penile erection in streptozotocin-induced diabetic rat]. | to determine whether recombinant adeno-associated virus-mediated overexpression of hcgrp in the corpus cavernosum can affect the continuous production of hcgrp in the penile tissue and enhance erectile responses in stz-induced diabetic rats. | 2005 | 16281515 |
| molecular characterization of adeno-associated viruses infecting children. | although adeno-associated virus (aav) infection is common in humans, the biology of natural infection is poorly understood. since it is likely that many primary aav infections occur during childhood, we set out to characterize the frequency and complexity of circulating aav isolates in fresh and archived frozen human pediatric tissues. total cellular dna was isolated from 175 tissue samples including freshly collected tonsils (n = 101) and archived frozen samples representing spleen (n = 21), lu ... | 2005 | 16282478 |
| characterization of adeno-associated virus genomes isolated from human tissues. | infection with wild-type adeno-associated virus (aav) is common in humans, but very little is known about the in vivo biology of aav. on a molecular level, it has been shown in cultured cells that aav integrates in a site-specific manner on human chromosome 19, but this has never been demonstrated directly in infected human tissues. to that end, we tested 175 tissue samples for the presence of aav dna, and when present, examined the specific form of the viral dna. aav was detected in 7 of 101 to ... | 2005 | 16282479 |
| adenovirus, adeno-associated virus and kawasaki disease. | clinical similarities and shared seasonality suggested a relationship between adenovirus infection and kawasaki disease. we performed adenovirus serology and quantitative polymerase chain reaction for both adenovirus and adeno-associated virus in patients with acute kawasaki disease. no evidence was found to suggest a link between either virus and kawasaki disease. | 2005 | 16282942 |
| adeno-associated vector mediated gene transfer of transforming growth factor-beta1 to normal and osteoarthritic human chondrocytes stimulates cartilage anabolism. | the objective of the present study was to investigate whether cartilage anabolism in human primary osteoarthritic chondrocytes could be improved by adeno-associated virus (aav) vector-mediated gene transduction of transforming growth factor tgf-beta1 (tgf-beta1). a bi-cistronic aav-tgf-beta1-ires-egfp (aav-tgf-beta1) vector was generated and used for transduction of a normal human articular chondrocyte cell line (tst/ac62) and primary human osteoarthritic articular chondrocytes harvested from 8 ... | 2005 | 16284937 |
| gene transfer of human acid sphingomyelinase corrects neuropathology and motor deficits in a mouse model of niemann-pick type a disease. | niemann-pick type a disease is a lysosomal storage disorder caused by a deficiency in acid sphingomyelinase (asm) activity. previously we showed that storage pathology in the asm knockout (asmko) mouse brain can be corrected by adeno-associated virus serotype 2 (aav2)-mediated gene transfer. the present experiment compared the relative therapeutic efficacy of different recombinant aav serotype vectors (1, 2, 5, 7, and 8) using histological, biochemical, and behavioral endpoints. in addition, we ... | 2005 | 16301517 |
| biological approaches to bone regeneration by gene therapy. | safe, effective approaches for bone regeneration are needed to reverse bone loss caused by trauma, disease, and tumor resection. unfortunately, the science of bone regeneration is still in its infancy, with all current or emerging therapies having serious limitations. unlike current regenerative therapies that use single regenerative factors, the natural processes of bone formation and repair require the coordinated expression of many molecules, including growth factors, bone morphogenetic prote ... | 2005 | 16304438 |
| adeno-associated virus-based gene therapy for inherited disorders. | adeno-associated virus vectors are capable of long-term gene transfer without obvious adverse effects in a number of animal models. over the last two decades, preclinical and early phase clinical trials in cystic fibrosis and alpha-1 antitrypsin deficiency were undertaken to test the feasibility of this approach. the results of those studies have been important since they have indicated that in vivo gene transfer is feasible and relatively safe. in addition, a number of key limitations to the cu ... | 2005 | 16306183 |
| novel caprine adeno-associated virus (aav) capsid (aav-go.1) is closely related to the primate aav-5 and has unique tropism and neutralization properties. | preexisting humoral immunity to adeno-associated virus (aav) vectors may limit their clinical utility in gene delivery. we describe a novel caprine aav (aav-go.1) capsid with unique biological properties. aav-go.1 capsid was cloned from goat-derived adenovirus preparations. surprisingly, aav-go.1 capsid was 94% identical to the human aav-5, with differences predicted to be largely on the surface and on or under the spike-like protrusions. in an in vitro neutralization assay using human immunoglo ... | 2005 | 16306595 |
| effects of epoxyeicosatrienoic acids on levels of enos phosphorylation and relevant signaling transduction pathways involved. | endothelial nitric oxide synthase (enos) is a key enzyme responsible for the regulation of vascular homeostasis. many humor factors and mechanical forces can affect enos activity via phosphorylation modification but the mechanisms involved vary with stimuli applied. we have demonstrated that cytochrome p450 (cyp) epoxygenase-dependent metabolites of arachidonic acid, epoxyeicosatrienoic acids (eets), can robustly up-regulate enos expression and its activity, however the relevant signaling pathwa ... | 2005 | 16315601 |
| oral adeno-associated virus-strail gene therapy suppresses human hepatocellular carcinoma growth in mice. | the extracellular domain of the tumor necrosis factor-related apoptosis-inducing ligand (strail) may function as a soluble cytokine to selectively kill various cancer cells without toxicity to most normal cells. we constructed a series of recombinant adeno-associated virus (aav) vectors expressing the extracellular domain of human trail fused with signal peptides of human insulin, interferon, human growth hormone, and serum albumin and designated them as aav-isn-t, aav-ifn-t, aav-hgh-t, and aav- ... | 2005 | 16317690 |
| virus-based therapies for colon cancer. | viral vectors are under development for anticancer therapy. as they can infect tumours and activate the immune system, viral vectors may directly destroy cancers (oncolysis), deliver genes with antitumour activity directly to the cancer cells, or act as cancer vaccines. better insights into the biology of the various vectors in use (e.g., poxvectors, adenovirus, adeno-associated virus, reovirus, newcastle disease virus) are making it possible to engineer viruses that are more tumour-specific, ef ... | 2005 | 16318426 |
| pharmacological and raav gene therapy rescue of visual functions in a blind mouse model of leber congenital amaurosis. | leber congenital amaurosis (lca), a heterogeneous early-onset retinal dystrophy, accounts for approximately 15% of inherited congenital blindness. one cause of lca is loss of the enzyme lecithin:retinol acyl transferase (lrat), which is required for regeneration of the visual photopigment in the retina. | 2005 | 16250670 |
| il-10 suppresses chemokines, inflammation, and fibrosis in a model of chronic renal disease. | il-10 is a pluripotent cytokine that plays a pivotal role in the regulation of immune and inflammatory responses. whereas short-term administration of il-10 has shown benefit in acute glomerulonephritis, no studies have addressed the potential benefits of il-10 in chronic renal disease. chronically elevated blood levels of il-10 in rats were achieved by administration of a recombinant adeno-associated virus serotype 1 il-10 (raav1-il-10) vector. control rats were given a similar dose of raav1-gf ... | 2005 | 16251240 |
| [gene transfer patterns and transduction efficacy of recombinant adeno-associated virus type 1, 2, and 5 in brain: an experiment with rats]. | to investigate the gene transfer patterns and transduction efficacy of different serotypes of recombinant adeno-associated virus (raav): raav1, 2, and 5 in brain. | 2005 | 16321184 |
| prevention and reversal of lupus in nzb/nzw mice by costimulatory blockade with adeno-associated virus-mediated gene transfer. | to investigate the potency of costimulatory blockade with adeno-associated virus (aav)-mediated gene transfer in the prevention and reversal of lupus in a murine model. | 2005 | 16329128 |
| [construction of recombinant adeno-associated virus vectors carrying double gene of antisense multidrug resistance-associated protein and antisense multidrug resistance]. | to construct a recombinant adeno-associated virus vectors carrying double gene of antisense multidrug resistance-associated protein (mrp) and antisense multidrug resistance (mdr1) for use in studying the gene therapy to reverse the multidrug resistance (mdr) in hepatocellular carcinoma (hcc). | 2005 | 16334547 |
| tendon healing in vitro: bfgf gene transfer to tenocytes by adeno-associated viral vectors promotes expression of collagen genes. | adeno-associated virus-mediated gene transfer is promising in the delivery of genes to tendons because this vector stimulates few adverse tissue reactions. basic fibroblast growth factor (bfgf) promotes collagen production in healing tendons. we transferred the exogenous bfgf gene to proliferating tenocytes by adeno-associated viral (aav) vectors and investigated its effects on the expression of the collagen genes in an in vitro tenocyte model. | 2005 | 16344185 |
| analysis of adeno-associated virus and hpv interaction. | it is slowly becoming accepted that adeno-associated virus (aav) is another significant factor involved in cervical carcinogenesis. however, unlike human papillomavirus (hpv), which is positively associated with cervical cancer, aav is negatively associated with this cancer. this negative association appears to be through a direct and complex bi-directional interaction between aav and hpv. essentially all assays used for studying hpv can be used for studying the aav-hpv interaction. this is beca ... | 2005 | 16350413 |
| [expression and effect of hcgrp recombinant adeno-associated virus in primary cultured corporal cavernosum smooth muscle cells of the rat in vitro]. | to observe the expression and effect of human calcitonin gene-related peptide (hcgrp) gene mediated by recombinant adeno-associated virus (raav) in primary cultured corporal cavernosum smooth muscle cells of the rat and explore the possibility of using cgrp gene for gene therapy in erectile dysfunction. | 2005 | 16398364 |
| the distribution, concentration, and toxicity of enhanced green fluorescent protein in retinal cells after genomic or somatic (virus-mediated) gene transfer. | the concentration of enhanced green fluorescent protein (egfp) in individual photoreceptor cells of live mouse retina was quantified and correlated with physiological measurements of cell function. | 2005 | 16402024 |
| [abeta vaccine therapy for alzheimer's disease]. | in 1999, schenk et al reported that vaccination of pdapp-transgenic mice with synthetic abeta42 in complete freund's adjuvant showed markedly decrease of the amyloid burden in the brain. the second trial of vaccine, an1792, for alzheimer's patients was halted because of meningoencephalitis found in 6% of patients and one autopsy case was reported. here, we comment the methods and the immunological mechanisms of abeta vaccine therapy and discuss the pathological changes in the brain and side effe ... | 2005 | 16447748 |
| repair of articular cartilage defect by intraarticular administration of basic fibroblast growth factor gene, using adeno-associated virus vector. | the objective of this study was to establish the potency of adeno-associated virus (aav) as a viral vector to transport the basic fibroblast growth factor (bfgf) gene into synovial tissue, and to evaluate the consequent repair of articular cartilage defects. in the in vitro study, lacz- and bfgf-encoding genes were transduced into rabbit synoviocytes by recombinant adeno-associated virus (aav) vector, and the cells were cultured for 2 weeks. the percentage of successfully transduced lacz-positiv ... | 2005 | 16390272 |