Thursday, November 7, 2013
This week in techniques
Publication and contact
Site-directed mRNA editing
to correct genetic diseases
A method to
site-specifically edit mRNA could be used to correct genetic mutations
associated with inherited diseases. A human adenosine
deaminase catalytic domain was fused to an RNA-binding protein
and then expressed in cells in conjunction with an antisense RNA that the
fusion protein recruits to the target mRNA sequence. In Xenopus
extracts, injection of RNA encoding the fusion protein and an antisense RNA
that targets a mutant form of the cystic fibrosis transmembrane conductance regulator
(CFTR) corrected 20% of the
transcripts on average. In human cells, transfection of multiple vectors
encoding the system enabled the correction of a mutant form of GFP. Next
steps include increasing the efficiency of the approach and testing it in
zebrafish and mouse models.
The study is funded by a 2013 NIH
transformative R01 award.
Published online Nov. 7, 2013
Patent application filed;
licensing status undisclosed
Montiel-Gonzalez, M.F. et
al. Proc. Natl. Acad. Sci. USA;
published online Oct. 9, 2013;
Contact: Joshua J.C. Rosenthal, University of Puerto Rico-Medical
Sciences Campus, San Juan,
All contents Copyright © 1993-2016 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 ]