Spell Checking Nature: Versatility of CRISPR/Cas9 for Developing Treatments for Inherited Disorders

Clustered regularly interspaced short palindromic repeat (CRISPR) has arisen as a frontrunner for efficient genome engineering. However, the potentially broad therapeutic implications are largely unexplored. Here, to investigate the therapeutic potential of CRISPR/Cas9 in a diverse set of genetic disorders, we establish a pipeline that uses readily obtainable cells from affected individuals. We show that an adapted version of CRISPR/Cas9 increases the amount of utrophin, a known disease modifier in Duchenne muscular dystrophy (DMD).

Source: The American Journal of Human Genetics - December 10, 2015 Category: Genetics & Stem Cells Authors: Daria Wojtal, Dwi U. Kemaladewi, Zeenat Malam, Sarah Abdullah, Tatianna W.Y. Wong, Elzbieta Hyatt, Zahra Baghestani, Sergio Pereira, James Stavropoulos, Vincent Mouly, Kamel Mamchaoui, Francesco Muntoni, Thomas Voit, Hernan D. Gonorazky, James J. Dowl Tags: Article Source Type: research