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 and Dentistry

Neuroscience

Keyphrases

Biochemistry, Genetics and Molecular Biology