This week in techniques



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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.

SciBX 6(43); doi:10.1038/scibx.2013.1242
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,
Puerto Rico