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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Bibliografiske detaljer
Udgivet i: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.
Format: Artigo
Sprog:Inglês
Udgivet: American Society of Gene & Cell Therapy 2017
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Original Article
Online adgang: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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