RNA editing for MECP2 mutations via RESTORE

Thorsten Stafforst, PhD

$366,520 AWARDED

RNA editing is a major focus of RSRT with currently funded efforts underway in the laboratories of Gail Mandel, Peter Beal, Guoping Feng and Jonathan Watts. We are pleased to now add Thorsten Stafforst, a pioneer in the field of RNA editing.

Dr. Stafforst has developed a simple, efficient and specific RNA editing platform called RESTORE which hijacks the RNA editing enzyme, ADAR, with a small piece of DNA or RNA called an antisense oligonucleotide (ASO).  ASOs don’t require a delivery vehicle and are likely to be less immunogenic and have improved biodistribution in the brain compared to viral vectors used to deliver other types of therapeutic molecules.

 

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