Development of an optimized AAV2/5 gene therapy vector for Leber congenital amaurosis owing to defects in RPE65

Results at 2 Years after Gene Therapy for RPE65-Deficient Leber Congenital Amaurosis and Severe Early-Childhood–Onset Retinal Dystrophy

(2016) Richard G. Weleber et al.   OPHTHALMOLOGY

Decoding mechanisms by which silent codon changes influence protein biogenesis and function

(2015) Vedrana Bali et al.   INTERNATIONAL JOURNAL OF BIOCHEMISTRY & CELL BIOLOGY

Gene Augmentation Therapy Restores Retinal Function and Visual Behavior in a Sheep Model of CNGA3 Achromatopsia

(2015) Eyal Banin et al.   MOLECULAR THERAPY

Long-Term Effect of Gene Therapy on Leber’s Congenital Amaurosis

(2015) James W.B. Bainbridge et al.   NEW ENGLAND JOURNAL OF MEDICINE

Improvement and Decline in Vision with Gene Therapy in Childhood Blindness

(2015) Samuel G. Jacobson et al.   NEW ENGLAND JOURNAL OF MEDICINE

Gene therapy restores vision in rd1 mice after removal of a confounding mutation in Gpr179

(2015) Koji M. Nishiguchi et al.   Nature Communications

RPE65 gene therapy slows cone loss in Rpe65-deficient dogs

(2012) F M Mowat et al.   GENE THERAPY

Gene Therapy for Leber Congenital Amaurosis Caused by RPE65 Mutations

(2011) Samuel G. Jacobson   ARCHIVES OF OPHTHALMOLOGY

An alternative isomerohydrolase in the retinal Müller cells of a cone-dominant species

(2011) Yusuke Takahashi et al.   FEBS Journal

Optic Vesicle-like Structures Derived from Human Pluripotent Stem Cells Facilitate a Customized Approach to Retinal Disease Treatment

(2011) Jason S. Meyer et al.   STEM CELLS

Gene therapy in the second eye of RPE65-deficient dogs improves retinal function

(2010) M J Annear et al.   GENE THERAPY

Modeling early retinal development with human embryonic and induced pluripotent stem cells

(2009) J. S. Meyer et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Treatment of Leber Congenital Amaurosis Due to RPE65 Mutations by Ocular Subretinal Injection of Adeno-Associated Virus Gene Vector: Short-Term Results of a Phase I Trial

(2008) William W. Hauswirth et al.   HUMAN GENE THERAPY

Safety and Efficacy of Gene Transfer for Leber's Congenital Amaurosis

(2008) Albert M. Maguire et al.   NEW ENGLAND JOURNAL OF MEDICINE

Effect of Gene Therapy on Visual Function in Leber's Congenital Amaurosis

(2008) James W.B. Bainbridge et al.   NEW ENGLAND JOURNAL OF MEDICINE

Leber congenital amaurosis: Genes, proteins and disease mechanisms

(2008) Anneke I. den Hollander et al.   PROGRESS IN RETINAL AND EYE RESEARCH