Biology and therapy of inherited retinal degenerative disease: insights from mouse models.

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Published in Dis Model Mech on February 01, 2015

Authors

Shobi Veleri1, Csilla H Lazar2, Bo Chang3, Paul A Sieving4, Eyal Banin5, Anand Swaroop6

Author Affiliations

1: Neurobiology-Neurodegeneration and Repair Laboratory, National Eye Institute, National Institutes of Health, Bethesda, MD 20892, USA.
2: Neurobiology-Neurodegeneration and Repair Laboratory, National Eye Institute, National Institutes of Health, Bethesda, MD 20892, USA. Molecular Biology Center, Interdisciplinary Research Institute on Bio-Nano Sciences, Babes-Bolyai-University, Cluj-Napoca, 400271, Romania.
3: The Jackson Laboratory, Bar Harbor, ME 04609, USA.
4: National Eye Institute, National Institutes of Health, Bethesda, MD 20892, USA.
5: Neurobiology-Neurodegeneration and Repair Laboratory, National Eye Institute, National Institutes of Health, Bethesda, MD 20892, USA. Center for Retinal and Macular Degenerations, Department of Ophthalmology, Hadassah-Hebrew University Medical Center, Jerusalem 91120, Israel.
6: Neurobiology-Neurodegeneration and Repair Laboratory, National Eye Institute, National Institutes of Health, Bethesda, MD 20892, USA. swaroopa@nei.nih.gov.

Associated clinical trials:

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

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 Study of an Adeno-associated Virus Vector for Gene Therapy of Leber's Hereditary Optic Neuropathy (LHON) | NCT02161380

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