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Strong ubiquitous micro-promoters for recombinant adeno-associated viral vectors

Significant progress has been made in developing recombinant adeno-associated virus (rAAV) for clinical gene therapy. While rAAV is a versatile gene delivery platform, its packaging limit of 4.7 kb limits the diseases it can target. Here, we report two unusually small promoters that enable the expre...

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Detalles Bibliográficos
Autores principales: Chai, Sunghee, Wakefield, Leslie, Norgard, Mason, Li, Bin, Enicks, David, Marks, Daniel L., Grompe, Markus
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10241652/
https://www.ncbi.nlm.nih.gov/pubmed/37287749
http://dx.doi.org/10.1016/j.omtm.2023.05.013