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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

1 Sept 2014

Abstract excerpt

A recombinant virus vector constructed from adeno-asso-ciated virus (AAV) has been engineered to carry the humanacid alpha-glucosidase (hGAA) gene expressed from thecytomegalovirus (CMV) enhancer/promoter. The constructhas been shown to produce hGAA in animal models ofPompe disease, which directly matches the proposed humanstudies. The proposed clinical trial is an open label, phase I/II study administering...

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PMID: 25238277
DOI: 10.1089/humc.2014.2514

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