Phase I/II Trial of Diaphragm Delivery of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase (rAAV1-CMV-GAA) Gene Vector in Patients with Pompe Disease

HUMAN GENE THERAPY CLINICAL DEVELOPMENT

卷 25, 期 3, 页码 134-163

MARY ANN LIEBERT, INC

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Biotechnology & Applied Microbiology Critical Care Medicine Medicine, Research & Experimental