Pre-clinical Safety and Off-Target Studies to Support Translation of AAV-Mediated RNAi Therapy for FSHD

RNAi emerged as a prospective molecular therapy nearly 15 years ago. Since then, two major RNAi platforms have been under development: oligonucleotides and gene therapy. Oligonucleotide-based approaches have seen more advancement, with some promising therapies that may soon reach market. In contrast...

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Detalles Bibliográficos
Autores principales: Wallace, Lindsay M., Saad, Nizar Y., Pyne, Nettie K., Fowler, Allison M., Eidahl, Jocelyn O., Domire, Jacqueline S., Griffin, Danielle A., Herman, Adam C., Sahenk, Zarife, Rodino-Klapac, Louise R., Harper, Scott Q.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2017
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5787672/
https://www.ncbi.nlm.nih.gov/pubmed/29387734
http://dx.doi.org/10.1016/j.omtm.2017.12.005

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