Vector platforms for gene therapy of inherited retinopathies.

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🔗 View Article (PMC 4241499)

Published in Prog Retin Eye Res on August 12, 2014

Authors

Ivana Trapani1, Agostina Puppo1, Alberto Auricchio2

Author Affiliations

1: Telethon Institute of Genetics and Medicine (TIGEM), Naples, Italy.
2: Telethon Institute of Genetics and Medicine (TIGEM), Naples, Italy; Medical Genetics, Department of Translational Medicine, Federico II University, Naples, Italy. Electronic address: auricchio@tigem.it.

Associated clinical trials:

Safety Study in Subjects With Leber Congenital Amaurosis | NCT00516477

Phase I Trial of Gene Vector to Patients With Retinal Disease Due to RPE65 Mutations (LCA) | NCT00481546

Safety Study of RPE65 Gene Therapy to Treat Leber Congenital Amaurosis | NCT00643747

Phase I/IIa Study of SAR422459 in Patients With Stargardt's Macular Degeneration | NCT01367444

Safety and Efficacy Study of rAAV2-ND4 Treatment of Leber Hereditary Optic Neuropathy (LHON) (rAAV2-ND4) | NCT01267422

Gene Therapy for Blindness Caused by Choroideremia | NCT01461213

Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis | NCT00999609

Study of UshStat in Patients With Retinitis Pigmentosa Associated With Usher Syndrome Type 1B | NCT01505062

Trial of Ocular Subretinal Injection of a Recombinant Adeno-Associated Virus (rAAV2-VMD2-hMERTK) Gene Vector to Patients With Retinal Disease Due to MERTK Mutations | NCT01482195

Safety Evaluation of Gene Therapy in Leber Hereditary Optic Neuropathy (LHON) Patients | NCT02064569

Clinical Gene Therapy Protocol for the Treatment of Retinal Dystrophy Caused by Defects in RPE65 (RPE65) | NCT01496040

Evaluation of Safety of Ciliary Neurotrophic Factor Implants in the Eye | NCT00063765

A Study to Determine the Long Term Safety, Tolerability and Biological Activity of SAR422459 in Patients With Stargardt's Macular Degeneration | NCT01736592

A Study to Determine the Long-Term Safety, Tolerability and Biological Activity of UshStat® in Patients With Usher Syndrome Type 1B | NCT02065011

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