419 Efficient CRISPR/Cas9-mediated knockout of Col7a1 in NOD/SCID IL2Rgamma-null mice by pro-nuclear injection

Current immunocompetent Col7A1-null mouse models of recessive dystrophic epidermolysis bullosa (RDEB) are not amenable to studies involving the transplantation of human cells. To address this limitation, we used pro-nuclear injection and CRISPR/Cas9 technology to disrupt Col7a1 in immunodeficient NOD/SCID IL2Rg-null (NSG) embryos; a strain permissive to adoptive transfer of human cells. Tandem guide RNAs (gRNA) targeting exon two of Col7a1 were co-delivered with Cas9 into single-cell embryos via pronuclear injection.

Source: Journal of Investigative Dermatology - April 20, 2016 Category: Dermatology Authors: B. Webber, K. O’Connor, R.T. McElmurry, E.N. Durgin, M.J. Riddle, M.J. Osborn, J. Tolar Tags: Genetic Disease, Gene Regulation & Gene Therapy Source Type: research