Preclinical development of , an investigational liver-directed AAV gene therapy for the treatment of Pompe disease
Sean M. Armour(Spark Therapeutics (United States)), Federico Mingozzi(Spark Therapeutics (United States)), Xavier M. Anguela(Children's Hospital of Philadelphia), Marco Crosariol(Spark Therapeutics (United States)), Christopher Riling(Spark Therapeutics (United States)), Helena Costa Verdera(Spark Therapeutics (United States)), Fanny Collaud(Inserm), Giuseppe Ronzitti(Inserm), Virginia Haurigot(Spark Therapeutics (United States)), Pauline Sellier(Inserm), George M. Preston(Spark Therapeutics (United States)), Pasqualina Colella(Stanford Medicine), William J. Quinn(University of Pennsylvania), Francesco Puzzo(Inserm), Umut Cagin(Inserm), Daniel M. Cohen, Hayley A. Hanby(Spark Therapeutics (United States)), Jayme M.L. Nordin(Spark Therapeutics (United States))
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