Human retinal gene therapy for Leber congenital amaurosis shows advancing retinal degeneration despite enduring visual improvement

Neuroprotective Strategies for the Treatment of Inherited Photoreceptor Degeneration

(2012) D. Trifunovic et al.   CURRENT MOLECULAR MEDICINE

Gene therapy restores vision and delays degeneration in the CNGB1-/- mouse model of retinitis pigmentosa

(2012) S. Koch et al.   HUMAN MOLECULAR GENETICS

Long-term expression of glial cell line-derived neurotrophic factor slows, but does not stop retinal degeneration in a model of retinitis pigmentosa

(2012) Masayuki Ohnaka et al.   JOURNAL OF NEUROCHEMISTRY

Differential Proteomics and Functional Research following Gene Therapy in a Mouse Model of Leber Congenital Amaurosis

(2012) Qinxiang Zheng et al.   PLoS One

Gene therapy rescues photoreceptor blindness in dogs and paves the way for treating human X-linked retinitis pigmentosa

(2012) W. A. Beltran et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Gene Therapy for Leber Congenital Amaurosis Caused by RPE65 Mutations

(2011) Samuel G. Jacobson   ARCHIVES OF OPHTHALMOLOGY

Early apoptosis of rod photoreceptors in Rpe65−/− mice is associated with the upregulated expression of lysosomal-mediated autophagic genes

(2011) Sylviane Métrailler et al.   EXPERIMENTAL EYE RESEARCH

Chemistry and Biology of Vision

(2011) Krzysztof Palczewski   JOURNAL OF BIOLOGICAL CHEMISTRY

AAV Mediated GDNF Secretion From Retinal Glia Slows Down Retinal Degeneration in a Rat Model of Retinitis Pigmentosa

(2011) Deniz Dalkara et al.   MOLECULAR THERAPY

Gene Therapy Regenerates Protein Expression in Cone Photoreceptors in Rpe65R91W/R91W Mice

(2011) Corinne Kostic et al.   PLoS One

Gene therapy prevents photoreceptor death and preserves retinal function in a Bardet-Biedl syndrome mouse model

(2011) D. L. Simons et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

The Genomic, Biochemical, and Cellular Responses of the Retina in Inherited Photoreceptor Degenerations and Prospects for the Treatment of These Disorders

(2010) Alexa N. Bramall et al.   Annual Review of Neuroscience

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

(2010) M J Annear et al.   GENE THERAPY

Replacement Gene Therapy with a Human RPGRIP1 Sequence Slows Photoreceptor Degeneration in a Murine Model of Leber Congenital Amaurosis

(2010) Basil S. Pawlyk et al.   HUMAN GENE THERAPY

Molecular Anthropology Meets Genetic Medicine to Treat Blindness in the North African Jewish Population: Human Gene Therapy Initiated in Israel

(2010) Eyal Banin et al.   HUMAN GENE THERAPY

Gene therapy rescues cone function in congenital achromatopsia

(2010) András M. Komáromy et al.   HUMAN MOLECULAR GENETICS

Long-term Retinal Function and Structure Rescue Using Capsid Mutant AAV8 Vector in the rd10 Mouse, a Model of Recessive Retinitis Pigmentosa

(2010) Ji-jing Pang et al.   MOLECULAR THERAPY

Restoration of visual function in P23H rhodopsin transgenic rats by gene delivery of BiP/Grp78

(2010) M. S. Gorbatyuk et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Leber congenital amaurosis due to RPE65 mutations and its treatment with gene therapy

(2010) Artur V. Cideciyan   PROGRESS IN RETINAL AND EYE RESEARCH

Human RPE65 Gene Therapy for Leber Congenital Amaurosis: Persistence of Early Visual Improvements and Safety at 1 Year

(2009) Artur V. Cideciyan et al.   HUMAN 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

Loss of cone photoreceptors caused by chromophore depletion is partially prevented by the artificial chromophore pro-drug, 9-cis-retinyl acetate

(2009) T. Maeda et al.   HUMAN MOLECULAR GENETICS

Bax-Induced Apoptosis in Leber's Congenital Amaurosis: A Dual Role in Rod and Cone Degeneration

(2009) Séverine Hamann et al.   PLoS One

Triggering of Bcl-2-related pathway is associated with apoptosis of photoreceptors in Rpe65 −/− mouse model of Leber’s Congenital Amaurosis

(2008) Sandra Cottet et al.   APOPTOSIS

ABCA4 disease progression and a proposed strategy for gene therapy

(2008) Artur V. Cideciyan et al.   HUMAN MOLECULAR GENETICS

Long-term effects of Aβ42 immunisation in Alzheimer's disease: follow-up of a randomised, placebo-controlled phase I trial

(2008) Clive Holmes et al.   LANCET

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