Functional and Behavioral Restoration of Vision by Gene Therapy in the Guanylate Cyclase-1 (GC1) Knockout Mouse

Gene therapy with a promoter targeting both rods and cones rescues retinal degeneration caused by AIPL1 mutations

(2009) X Sun et al.   GENE THERAPY

Gene therapy for retinitis pigmentosa and Leber congenital amaurosis caused by defects in AIPL1: effective rescue of mouse models of partial and complete Aipl1 deficiency using AAV2/2 and AAV2/8 vectors

(2009) Mei Hong Tan et al.   HUMAN MOLECULAR GENETICS

Leber congenital amaurosis: Clinical correlations with genotypes, gene therapy trials update, and future directions

(2009) Daniel C. Chung et al.   JOURNAL OF AAPOS

A Functional Kinase Homology Domain Is Essential for the Activity of Photoreceptor Guanylate Cyclase 1

(2009) Grzegorz Bereta et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Novel functions of photoreceptor guanylate cyclases revealed by targeted deletion

(2009) Sukanya Karan et al.   MOLECULAR AND CELLULAR BIOCHEMISTRY

Gene therapy for red–green colour blindness in adult primates

(2009) Katherine Mancuso et al.   NATURE

Vision 1 Year after Gene Therapy for Leber's Congenital Amaurosis

(2009) Artur V. Cideciyan et al.   NEW ENGLAND JOURNAL OF MEDICINE

Photoreceptor IFT Complexes Containing Chaperones, Guanylyl Cyclase 1 and Rhodopsin

(2009) Reshma Bhowmick et al.   TRAFFIC

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

Speed, Spatial, and Temporal Tuning of Rod and Cone Vision in Mouse

(2008) Y. Umino et al.   JOURNAL OF NEUROSCIENCE

Subretinal Delivery of Recombinant AAV Serotype 8 Vector in Dogs Results in Gene Transfer to Neurons in the Brain

(2008) Knut Stieger et al.   MOLECULAR 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

Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics

(2008) A. V. Cideciyan et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Leber congenital amaurosis: Genes, proteins and disease mechanisms

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