Intercellular Transfer of the Virally Derived Precursor Form of Acid α-Glucosidase Corrects the Enzyme Deficiency in Inherited Cardioskeletal Myopathy Pompe Disease

Daniel Pauly(University of British Columbia), Barry J. Byrne(University of Florida), Helen S. Bayes(Johns Hopkins University), Paul H. Plötz(National Institutes of Health), Nina Raben(APT Therapeutics (United States)), Thomas J. Fraites(University of Florida), Maryann L. Huie(Columbia University Irving Medical Center), Paul Kessler(Johns Hopkins University), Rochelle Hirschhorn(New York University), Catalin Toma(University of Pittsburgh Medical Center)
Human Gene Therapy
March 20, 2001
Cited by 50


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