CORDIS Project
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This project aims to develop a gene therapy approach for treating limb-girdle muscular dystrophy type 2A by correcting a specific mutation in patient-derived stem cells. The research includes testing the therapeutic potential in mouse models as a precursor to clinical trials.
In Duchenne muscular dystrophy, several mutations can be corrected by the exon-skipping technique on cells.
In limb-girdle muscular dystrophy type 2A (LGMD2A), however, all the mutations described to date do not seem to be good candidates for the RNA correction technology.
The recently identified c.1782+1072 G>C mutation in LGMD2A patients could be potentially repaired at RNA level.
The principal aim of this project is to mask the effect of this mutation in stem cells from patients that can be d…
ASOCIACION INSTITUTO BIOGIPUZKOA
ASOCIACION INSTITUTO DE INVESTIGACION SANITARIA BIOGIPUZKOA
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