Optimization of systemic AAV9 gene therapy in Niemann-Pick disease type C1 mice.

Autor: Mylvara AV; Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, Department of Human Health and Services, Bethesda, MD.; National Human Genome Research Institute, National Institutes of Health, Department of Human Health and Services, Bethesda, MD., Gibson AL; National Human Genome Research Institute, National Institutes of Health, Department of Human Health and Services, Bethesda, MD.; Howard Hughes Medical Institute, Department of Cellular and Molecular Medicine, Section of Neurobiology, Division of Biological Sciences, University of California, San Diego, San Diego, CA., Gu T; National Human Genome Research Institute, National Institutes of Health, Department of Human Health and Services, Bethesda, MD.; University of North Carolina, Chapel Hill, NC., Davidson CD; Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, Department of Human Health and Services, Bethesda, MD.; National Human Genome Research Institute, National Institutes of Health, Department of Human Health and Services, Bethesda, MD., Incao AA; National Human Genome Research Institute, National Institutes of Health, Department of Human Health and Services, Bethesda, MD., Melnyk K; Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, Department of Human Health and Services, Bethesda, MD., Pierre-Jacques D; University of Illinois Chicago, Chicago, IL., Cologna SM; University of Illinois Chicago, Chicago, IL., Venditti CP; National Human Genome Research Institute, National Institutes of Health, Department of Human Health and Services, Bethesda, MD., Porter FD; Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, Department of Human Health and Services, Bethesda, MD., Pavan WJ; National Human Genome Research Institute, National Institutes of Health, Department of Human Health and Services, Bethesda, MD.

Publikováno v: BioRxiv : the preprint server for biology [bioRxiv] 2024 Jun 08. Date of Electronic Publication: 2024 Jun 08.