Article
Spell Checking Nature: Versatility of CRISPR/Cas9 for Developing Treatments for Inherited Disorders.
American journal of human genetics - 7 Jan 2016
Wojtal Daria, Kemaladewi Dwi U, Malam Zeenat, Abdullah Sarah, Wong Tatianna W Y, Hyatt Elzbieta, Baghestani Zahra, Pereira Sergio, Stavropoulos James, Mouly Vincent, Mamchaoui Kamel, Muntoni Francesco, Voit Thomas, Gonorazky Hernan D, Dowling James J, Wilson Michael D, Mendoza-Londono Roberto, Ivakine Evgueni A, Cohn Ronald D
Abstract excerpt
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...
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