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

• Главные авторы: Da Sun (Автор), Wenyu Sun (Автор), Song-Qi Gao (Автор), Jonathan Lehrer (Автор), Amirreza Naderi (Автор), Cheng Wei (Автор), Sangjoon Lee (Автор), Andrew L. Schilb (Автор), Josef Scheidt (Автор), Ryan C. Hall (Автор), Elias I. Traboulsi (Автор), Krzysztof Palczewski (Автор), Zheng-Rong Lu (Автор)
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• Опубликовано: Elsevier, 2022-09-01T00:00:00Z.
• Предметы: article
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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...