Intracranial delivery of AAV9 gene therapy partially prevents retinal degeneration and visual deficits in CLN6-Batten disease mice.

White, Katherine A; Nelvagal, Hemanth R; Poole, Timothy A; Lu, Bin; Johnson, Tyler B; Davis, Samantha; Pratt, Melissa A; Brudvig, Jon; Assis, Ana B; Likhite, Shibi; Meyer, Kathrin; Kaspar, Brian K; Cooper, Jonathan D; Wang, Shaomei; Weimer, Jill M

White, Katherine A; Nelvagal, Hemanth R; Poole, Timothy A; Lu, Bin; Johnson, Tyler B; Davis, Samantha; Pratt, Melissa A; Brudvig, Jon; Assis, Ana B; Likhite, Shibi; Meyer, Kathrin; Kaspar, Brian K; Cooper, Jonathan D; Wang, Shaomei; Weimer, Jill M.

Afiliação

White KA; Pediatrics and Rare Diseases Group, Sanford Research, Sioux Falls, SD 57104, USA.
Nelvagal HR; Pediatric Storage Disorders Laboratory, Division of Genetics and Genomics, Department of Pediatrics, Washington University School of Medicine, St. Louis, MO 63110, USA.
Poole TA; Department of Pediatrics, The Lundquist Institute at Harbor-UCLA Medical Center and David Geffen School of Medicine, UCLA, Torrance, CA 90502, USA.
Lu B; Pediatric Storage Disorders Laboratory, Division of Genetics and Genomics, Department of Pediatrics, Washington University School of Medicine, St. Louis, MO 63110, USA.
Johnson TB; Regenerative Medicine Institute, Cedars-Sinai Medical Center, Los Angeles, CA 90048, USA.
Davis S; Pediatrics and Rare Diseases Group, Sanford Research, Sioux Falls, SD 57104, USA.
Pratt MA; Amicus Therapeutics, Philadelphia, PA 19104, USA.
Brudvig J; Pediatrics and Rare Diseases Group, Sanford Research, Sioux Falls, SD 57104, USA.
Assis AB; Pediatrics and Rare Diseases Group, Sanford Research, Sioux Falls, SD 57104, USA.
Likhite S; Pediatrics and Rare Diseases Group, Sanford Research, Sioux Falls, SD 57104, USA.
Meyer K; Department of Pediatrics, The Lundquist Institute at Harbor-UCLA Medical Center and David Geffen School of Medicine, UCLA, Torrance, CA 90502, USA.
Kaspar BK; Nationwide Children's Hospital. He was involved in AAV9 construct development.
Cooper JD; The Research Institute at Nationwide Children's Hospital, Columbus, OH 43205, USA.
Wang S; Department of Pediatrics, The Ohio State University, Columbus, OH 43210, USA.
Weimer JM; The Research Institute at Nationwide Children's Hospital, Columbus, OH 43205, USA.