Gene Supplementation Rescues Rod Function and Preserves Photoreceptor and Retinal Morphology in Dogs, Leading the Way Toward Treating HumanPDE6A-Retinitis Pigmentosa-Reference-Cited by-同舟云学术

Gene Supplementation Rescues Rod Function and Preserves Photoreceptor and Retinal Morphology in Dogs, Leading the Way Toward Treating HumanPDE6A-Retinitis Pigmentosa

Published:2017-12 Issue:12 Volume:28 Page:1189-1201
ISSN:1043-0342
Container-title:Human Gene Therapy
language:en
Short-container-title:Human Gene Therapy

Author:

Occelli Laurence M.¹,Schön Christian²,Seeliger Mathias W.³,Biel Martin²,Michalakis Stylianos²,Petersen-Jones Simon M.¹,the RD-CURE Consortium

Affiliation:

1. Department of Small Animal Clinical Sciences, Michigan State University, East Lansing, Michigan.

2. Center for Integrated Protein Science Munich (CIPSM) at the Department of Pharmacy—Center for Drug Research, Ludwig-Maximilians-Universität München, Munich, Germany.

3. Division of Ocular Neurodegeneration, Institute for Ophthalmic Research, Centre for Ophthalmology, Eberhard Karls University, Tuebingen, Germany.

Publisher

Mary Ann Liebert Inc

Subject

Genetics,Molecular Biology,Molecular Medicine

Link

http://www.liebertpub.com/doi/pdf/10.1089/hum.2017.155

Reference47 articles.

1. Effect of Gene Therapy on Visual Function in Leber's Congenital Amaurosis

2. Safety and Efficacy of Gene Transfer for Leber's Congenital Amaurosis

3. Treatment of Leber Congenital Amaurosis Due toRPE65Mutations by Ocular Subretinal Injection of Adeno-Associated Virus Gene Vector: Short-Term Results of a Phase I Trial

4. The Status ofRPE65Gene Therapy Trials: Safety and Efficacy

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

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