Thursday, April 17, 2014
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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
and accumulation of toxic metabolites. A compound that acts upstream of FAH
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.
Published online April 17, 2014
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.
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