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Preclinical development of , an investigational liver-directed AAV gene therapy for the treatment of Pompe disease
Preclinical development of , an investigational liver-directed AAV gene therapy for the treatment of Pompe disease
2020
Sean M. Armour
Jayme M.L. Nordin
Helena Costa Verdera
Pauline Sellier
George M Preston
Christopher Riling
Daniel M. Cohen
Fanny Collaud
Marco Crosariol
William J. Quinn
Hayley Hanby
Umut Cagin
Francesco Puzzo
Virginia Haurigot
Giuseppe Ronzitti
Pasqualina Colella
Xavier M. Anguela
Federico Mingozzi
Keywords:
Genetics
Disease
Genetic enhancement
Diabetes mellitus
Bioinformatics
Biology
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