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Clustered, regularly interspaced short palindromic repeats (CRISPR)-based genome editing platform to treat genetic liver disease

Mouse studies suggest CRISPR-based genome editing could be used to treat tyrosinemia type I (TTI), a fatal disease resulting from mutation of fumarylacetoacetate hydrolase (FAH) and accumulation of toxic metabolites. A compound that acts upstream of FAH called 2-(2-nitro-4-trifluoromethlybenzoyl)-1,3-cyclohexanedione (NTBC) can keep Fah5981SB-mutant mice alive. In a Fah5981SB mouse model of hereditary TTI, tail vein injection of a Fah-correcting, single-stranded DNA (ssDNA) donor, a CRISPR-associated 9 (Cas9)-expressing ssDNA and a Fah-targeting single-guide RNA restored Fah mRNA to up to 36% of wild-type levels, resulted in widespread patches of Fah+ hepatocytes and prevented weight loss upon NTBC withdrawal. Next steps include testing the CRISPR platform in large-animal studies and developing delivery systems.

SciBX 7(15); doi:10.1038/scibx.2014.442
Published online April 17, 2014

Multiple patent applications filed; licensing discussions in progress

Yin, H. et al. Nat. Biotechnol.; published online March 30, 2014;
Contact: Daniel G. Anderson, Massachusetts Institute of Technology, Cambridge, Mass.