Toxicology Study of Intra-Cisterna Magna Adeno-Associated Virus 9 Expressing Iduronate-2-Sulfatase in Rhesus Macaques.

ABSTRACT: Hunter syndrome is an X-linked recessive disease caused by deficiency of the lysosomal enzyme iduronate-2-sulfatase. The severe form of this progressive, systemic, and neurodegenerative disease results in loss of cognitive skills and early death. Several clinical trials are evaluating adeno-associated virus 9 for the treatment of neurodegenerative diseases using systemic or intrathecal lumbar administration. In large animals, administration via suboccipital puncture gives better brain transduction than lumbar administration. Here, we conducted a good laboratory practice-compliant investigational new drug-enabling study to determine the safety of suboccipital adeno-associated virus 9 gene transfer of human iduronate-2-sulfatase into nonhuman primates. Thirteen rhesus macaques received vehic

SUBMITTER: Hordeaux J 

PROVIDER: S-EPMC6070702 | biostudies-literature | 2018 Sep

REPOSITORIES: biostudies-literature