Preclinical Potency and Safety Studies of an AAV2-Mediated Gene Therapy Vector for the Treatment of MERTK Associated Retinitis Pigmentosa-Reference-Cited by-同舟云学术

Preclinical Potency and Safety Studies of an AAV2-Mediated Gene Therapy Vector for the Treatment of MERTK Associated Retinitis Pigmentosa

Published:2013-03 Issue:1 Volume:24 Page:23-28
ISSN:2324-8637
Container-title:Human Gene Therapy Clinical Development
language:en
Short-container-title:Human Gene Therapy Clinical Development

Author:

Conlon Thomas J.¹,Deng Wen-Tao²,Erger Kirsten¹,Cossette Travis¹,Pang Ji-jing²,Ryals Renee²,Clément Nathalie¹,Cleaver Brian¹,McDoom Issam²,Boye Shannon E.²,Peden Marc C.²,Sherwood Mark B.²,Abernathy Corinne R.³,Alkuraya Fowzan S.⁴,Boye Sanford L.²,Hauswirth William W.²

Affiliation:

1. Powell Gene Therapy Center, Department of Pediatrics, University of Florida, Gainesville, FL 32610.

2. Department of Opthalmology, College of Medicine, University of Florida, Gainesville, FL 32610.

3. Clinical and Translational Science Institute (CTSI), University of Florida, Gainesville, FL 32610.

4. Department of Genetics, King Faisal Specialist Hospital & Research Centre, Riyadh, Saudi Arabia.

Publisher

Mary Ann Liebert Inc

Subject

Genetics (clinical)

Link

http://www.liebertpub.com/doi/pdf/10.1089/humc.2013.037

Reference26 articles.

1. Adeno-Associated Viral Vectors and the Retina

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

3. THE ROLE OF THE PIGMENT EPITHELIUM IN THE ETIOLOGY OF INHERITED RETINAL DYSTROPHY IN THE RAT

4. A Comprehensive Review of Retinal Gene Therapy

5. Characterisation of severe rod-cone dystrophy in a consanguineous family with a splice site mutation in the MERTK gene

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