Spliceosome-Mediated RNA Trans-Splicing Therapy and Protein Replacement in Rett Syndrome

Stuart Cobb, PhD | University of Glasgow

$627,012 AWARDED

Targeting MeCP2 can be done either at the DNA, mRNA or protein level. Stuart Cobb is working on all of these approaches in parallel. His work with gene therapy is being done as part of the Gene Therapy Consortium.

We know that both the DNA and protein approaches have a complication due to potential dosage problems (too much MeCP2 may be harmful). So Dr.Cobb is also pursuing an alternative technology called Spliceosome-Mediated RNA Trans-Splicing (SMaRT). This technology allows a mutation to be spliced out and repaired in RNA. The advantage is that this approach avoids any potential overexpression issues.

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