Effective gene therapy of Stargardt disease with PEG-ECO/pGRK1-ABCA4-S/MAR nanoparticles

Effective gene therapy of Stargardt disease with PEG-ECO/pGRK1-ABCA4-S/MAR nanoparticles

Stargardt disease (STGD) is the most common form of inherited retinal genetic disorders and is often caused by mutations in ABCA4. Gene therapy has the promise to effectively treat monogenic retinal disorders. However, clinically approved adeno-associated virus (AAV) vectors do not have a loading ca...

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Bibliographic Details
Main Authors: Da Sun, Wenyu Sun, Song-Qi Gao, Jonathan Lehrer, Amirreza Naderi, Cheng Wei, Sangjoon Lee, Andrew L. Schilb, Josef Scheidt, Ryan C. Hall, Elias I. Traboulsi, Krzysztof Palczewski, Zheng-Rong Lu
Format: Article
Language:English
Published: Elsevier 2022-09-01
Series:Molecular Therapy: Nucleic Acids
Subjects:
MT: Delivery strategies
non-viral gene therapy
Stargardt disease
ECO
ABCA4
nanoparticles
Online Access:http://www.sciencedirect.com/science/article/pii/S2162253122002256

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http://www.sciencedirect.com/science/article/pii/S2162253122002256

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