Initial results from a first-in-human gene therapy trial on X-linked retinitis pigmentosa caused by mutations in RPGR

Optical Coherence Tomography Angiography of RPGR-Associated Retinitis Pigmentosa Suggests Foveal Avascular Zone is a Biomarker for Vision Loss

(2019) Peter H. Tang et al.   Ophthalmic Surgery Lasers & Imaging Retina

Gene therapy and genome surgery in the retina

(2018) James E. DiCarlo et al.   JOURNAL OF CLINICAL INVESTIGATION

Retinal AAV8- RS1 Gene Therapy for X-Linked Retinoschisis: Initial Findings from a Phase I/IIa Trial by Intravitreal Delivery

(2018) Catherine Cukras et al.   MOLECULAR THERAPY

CLINICAL AND GENETIC CHARACTERISTICS OF MALE PATIENTS WITH RPGR-ASSOCIATED RETINAL DYSTROPHIES

(2018) Mays Talib et al.   RETINA-THE JOURNAL OF RETINAL AND VITREOUS DISEASES

Inherited Retinal Degenerations: Current Landscape and Knowledge Gaps

(2018) Jacque L. Duncan et al.   Translational Vision Science & Technology

Innovative Optogenetic Strategies for Vision Restoration

(2018) Cameron K. Baker et al.   Frontiers in Cellular Neuroscience

Technique of retinal gene therapy: delivery of viral vector into the subretinal space

(2017) K Xue et al.   EYE

Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65 -mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial

(2017) Stephen Russell et al.   LANCET

Taking Stock of Retinal Gene Therapy: Looking Back and Moving Forward

(2017) Jean Bennett   MOLECULAR THERAPY

Codon-Optimized RPGR Improves Stability and Efficacy of AAV8 Gene Therapy in Two Mouse Models of X-Linked Retinitis Pigmentosa

(2017) M. Dominik Fischer et al.   MOLECULAR THERAPY

Safety and durability of effect of contralateral-eye administration of AAV2 gene therapy in patients with childhood-onset blindness caused by RPE65 mutations: a follow-on phase 1 trial

(2016) Jean Bennett et al.   LANCET

Loss of RPGR glutamylation underlies the pathogenic mechanism of retinal dystrophy caused byTTLL5mutations

(2016) Xun Sun et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

RPGR: Its role in photoreceptor physiology, human disease, and future therapies

(2015) Roly D. Megaw et al.   EXPERIMENTAL EYE RESEARCH

Photoreceptor rescue by an abbreviated human RPGR gene in a murine model of X-linked retinitis pigmentosa

(2015) B S Pawlyk et al.   GENE THERAPY

Stability and Safety of an AAV Vector for TreatingRPGR-ORF15X-Linked Retinitis Pigmentosa

(2015) Wen-Tao Deng et al.   HUMAN GENE THERAPY

A long-term efficacy study of gene replacement therapy for RPGR-associated retinal degeneration

(2015) Zhijian Wu et al.   HUMAN MOLECULAR GENETICS

Rates of Decline in Regions of the Visual Field Defined by Frequency-Domain Optical Coherence Tomography in Patients with RPGR-Mediated X-Linked Retinitis Pigmentosa

(2015) David G. Birch et al.   OPHTHALMOLOGY

What Is Next for Retinal Gene Therapy?

(2015) Luk H. Vandenberghe   Cold Spring Harbor Perspectives in Medicine

Retinal gene therapy in patients with choroideremia: initial findings from a phase 1/2 clinical trial

(2014) Robert E MacLaren et al.   LANCET

Clinical Characteristics and Current Therapies for Inherited Retinal Degenerations

(2014) J.-A. Sahel et al.   Cold Spring Harbor Perspectives in Medicine

A Comprehensive Review of Retinal Gene Therapy

(2013) Shannon E Boye 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