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...

Disgrifiad llawn

Wedi'i Gadw mewn:
Manylion Llyfryddiaeth
Cyhoeddwyd yn:Mol Ther
Prif Awduron: 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.
Fformat: Artigo
Iaith:Inglês
Cyhoeddwyd: American Society of Gene & Cell Therapy 2017
Pynciau:
Original Article
Mynediad Ar-lein: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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