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Non-viral Gene Therapy for Stargardt Disease with ECO/pRHO-ABCA4 Self-Assembled Nanoparticles

  • Published:2020-01 Issue:1 Volume:28 Page:293-303
  • ISSN:1525-0016
  • Container-title:Molecular Therapy
  • language:en
  • Short-container-title:Molecular Therapy

Author:

Sun Da,Schur Rebecca M.,Sears Avery E.,Gao Song-Qi,Vaidya Amita,Sun Wenyu,Maeda Akiko,Kern Timothy,Palczewski Krzysztof,Lu Zheng-Rong

Funder

Harrington Discovery Institute

Foundation of Fighting Blindness

NEI

Canadian Institute for Advanced Research

Alcon Research Institute

Publisher

Elsevier BV

Subject

Drug Discovery,Pharmacology,Genetics,Molecular Biology,Molecular Medicine

Reference47 articles.

1. A comprehensive review of retinal gene therapy;Boye;Mol. Ther.,2013

2. Mutation of the Stargardt disease gene (ABCR) in age-related macular degeneration;Allikmets;Science,1997

3. The ATP-binding cassette transporter ABCA4: structural and functional properties and role in retinal disease;Tsybovsky,2010

4. Mechanism of all-trans-retinal toxicity with implications for stargardt disease and age-related macular degeneration;Chen;J. Biol. Chem.,2012

5. The gene for Stargardt disease, ABCA4, is a major retinal gene: a mini-review;Koenekoop;Ophthalmic Genet.,2003

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