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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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Detalles Bibliográficos
Autores principales:	Sun, Da, Sun, Wenyu, Gao, Song-Qi, Lehrer, Jonathan, Naderi, Amirreza, Wei, Cheng, Lee, Sangjoon, Schilb, Andrew L., Scheidt, Josef, Hall, Ryan C., Traboulsi, Elias I., Palczewski, Krzysztof, Lu, Zheng-Rong
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	American Society of Gene & Cell Therapy 2022
Materias:	Original Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9463552/ https://www.ncbi.nlm.nih.gov/pubmed/36159595 http://dx.doi.org/10.1016/j.omtn.2022.08.026

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