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,
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