Using CRISPR-Cas9 to Generate Gene-Corrected Autologous iPSCs for the Treatment of Inherited Retinal Degeneration

Patient-derived induced pluripotent stem cells (iPSCs) hold great promise for autologous cell replacement. However, for many inherited diseases, treatment will likely require genetic repair pre-transplantation. Genome editing technologies are useful for this application. The purpose of this study wa...

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Detalhes bibliográficos
Publicado no:Mol Ther
Main Authors: Burnight, Erin R., Gupta, Manav, Wiley, Luke A., Anfinson, Kristin R., Tran, Audrey, Triboulet, Robinson, Hoffmann, Jeremy M., Klaahsen, Darcey L., Andorf, Jeaneen L., Jiao, Chunhua, Sohn, Elliott H., Adur, Malavika K., Ross, Jason W., Mullins, Robert F., Daley, George Q., Schlaeger, Thorsten M., Stone, Edwin M., Tucker, Budd A.
Formato: Artigo
Idioma:Inglês
Publicado em: American Society of Gene & Cell Therapy 2017
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Original Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC5589061/
https://ncbi.nlm.nih.gov/pubmed/28619647
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2017.05.015
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