Thursday, April 17, 2014
Publication and contact
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.
All contents Copyright © 1993-2017 BioCentury Publications, Inc. ALL RIGHTS RESERVED. All use of this Web Site and its contents is governed by the BioCentury User Agreement
. The contents of this Web Site are protected under U.S. and foreign copyright and intellectual property laws, and no part of this Web Site or its contents may be photocopied, reproduced or retransmitted in any form without the written consent of BioCentury, which may be requested from Reprints/Permissions
. BioCentury®; The Bernstein Report on BioBusiness™; The BioCentury 100™; The Clear Route to ROI™; Because Real Intelligence is Hard to Find™; BCIQ™; and BioPharma's Knowledge Center™; are trademarks of BioCentury Publications, Inc., P.O. Box 1246, San Carlos, CA 94070. SciBX® and SciBX: Science-Business eXchange® are trademarks of Nature America, Inc. that are jointly used by BioCentury Publications, Inc. and Nature America, Inc.
Thanks for purchasing this article.
Please bookmark this url for future reference.
We have also sent you a confirming e-mail with a receipt and this same url for your
close [ X ]