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Current Clinical Applications of In Vivo Gene Therapy with AAVs

Hereditary diseases are caused by mutations in genes, and more than 7,000 rare diseases affect over 30 million Americans. For more than 30 years, hundreds of researchers have maintained that genetic modifications would provide effective treatments for many inherited human diseases, offering durable...

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Detalles Bibliográficos
Autores principales:	Mendell, Jerry R., Al-Zaidy, Samiah A., Rodino-Klapac, Louise R., Goodspeed, Kimberly, Gray, Steven J., Kay, Christine N., Boye, Sanford L., Boye, Shannon E., George, Lindsey A., Salabarria, Stephanie, Corti, Manuela, Byrne, Barry J., Tremblay, Jacques P.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	American Society of Gene & Cell Therapy 2021
Materias:	Review
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7854298/ https://www.ncbi.nlm.nih.gov/pubmed/33309881 http://dx.doi.org/10.1016/j.ymthe.2020.12.007

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