Gene Therapy Restores Vision-Dependent Behavior as Well as Retinal Structure and Function in a Mouse Model of RPE65 Leber Congenital Amaurosis

Gene therapy restores vision in a canine model of childhood blindness

Gregory M. Acland, Jharna Ray, Qí Zhāng et al. · Nature Genetics · 2001 · 1.2K citations

Medicine, Ophthalmology, Vision Science +16

Rpe65 is necessary for production of 11-cis-vitamin A in the retinal visual cycle

T. Michael Redmond, Shirley Yu, Eric Lee et al. · Nature Genetics · 1998 · 930 citations

Medicine, Ophthalmology, Vision Science +10

Novel Tools for Production and Purification of Recombinant Adenoassociated Virus Vectors

Dirk Grimm, Andrea Kern, Karola Rittner et al. · Human Gene Therapy · 1998 · 700 citations

Life Sciences, Biotechnology, Molecular Biology +16

Mutations in RPE65 cause autosomal recessive childhood–onset severe retinal dystrophy

Sumin Gu, Debra A. Thompson, C.R. Srikumari et al. · Nature Genetics · 1997 · 666 citations

Medicine, Genetics, Molecular Genetics +10

Mutations in the RPE65 gene in patients with autosomal recessive retinitis pigmentosa or Leber congenital amaurosis

Hiroyuki Morimura, Gerald A. Fishman, Sandeep Grover et al. · Proceedings of the National Academy of Sciences · 1998 · 489 citations

Medicine, Genetics, Molecular Genetics +15