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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Foilsithe in: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.
Formáid: Artigo
Teanga:Inglês
Foilsithe: American Society of Gene & Cell Therapy 2017
Ábhair:
Original Article
Rochtain Ar Líne: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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