Article
Using CRISPR-Cas9 to Generate Gene-Corrected Autologous iPSCs for the Treatment of Inherited Retinal Degeneration.
Molecular therapy : the journal of the American Society of Gene Therapy - 6 Sept 2017
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
Abstract excerpt
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 was to develop CRISPR-Cas9-mediated genome editing strategies to target and correct the three most common types of...
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