Adeno-associated virus (AAV)-mediated RNA interference to PABPN1 combined with an optimised resistant transgene for rescue of the muscle specific disease oculopharyngeal muscular dystrophy (OPMD)

Houria Bachtarzi, Alberto Malerba, Susan Jarmin, Capucine Trollet, Michael Graham, George Dickson

Research output: Contribution to conferenceAbstract

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Medicine & Life Sciences