Publications
| transduction of nonhuman primate brain with adeno-associated virus serotype 1: vector trafficking and immune response. | we used convection-enhanced delivery (ced) to characterize gene delivery mediated by adeno-associated virus type 1 (aav1) by tracking expression of hrgfp (humanized green fluorescent protein from renilla reniformis) into the striatum, basal forebrain, and corona radiata of monkey brain. four cynomolgus monkeys received single infusions into corona radiata, putamen, and caudate. the other group (n = 4) received infusions into basal forebrain. thirty days after infusion animals were killed and the ... | 2009 | 19292604 |
| broad distribution of ataxin 1 silencing in rhesus cerebella for spinocerebellar ataxia type 1 therapy. | spinocerebellar ataxia type 1 is one of nine polyglutamine expansion diseases and is characterized by cerebellar ataxia and neuronal degeneration in the cerebellum and brainstem. currently, there are no effective therapies for this disease. previously, we have shown that rna interference mediated silencing of atxn1 mrna provides therapeutic benefit in mouse models of the disease. adeno-associated viral delivery of an engineered microrna targeting atxn1 to the cerebella of well-established mouse ... | 2015 | 26490326 |