Shelley Collins

Phase I/II Trial of Adeno-Associated Virus–Mediated Alpha-Glucosidase Gene Therapy to the Diaphragm for Chronic Respiratory Failure in Pompe Disease: Initial Safety and Ventilatory Outcomes

Barbara K. Smith, Shelley Collins, Thomas J. Conlon et al. · Human Gene Therapy · 2013 · 154 citations · Full text

Acute Lung Injury, Pediatric Lung Disease, Chronic Respiratory Failure +16

Safety of Intradiaphragmatic Delivery of Adeno-Associated Virus-Mediated Alpha-Glucosidase (rAAV1-CMV-<i>hGAA</i>) Gene Therapy in Children Affected by Pompe Disease

Manuela Corti, Cristina Liberati, Barbara K. Smith et al. · Human Gene Therapy Clinical Development · 2017 · 112 citations · Full text

In Vivo Gene Therapy, Viral Pathogenesis, Immunology +16

B-Cell Depletion and Immunomodulation before Initiation of Enzyme Replacement Therapy Blocks the Immune Response to Acid Alpha-Glucosidase in Infantile-Onset Pompe Disease

Melissa E. Elder, Sushrusha Nayak, Shelley Collins et al. · The Journal of Pediatrics · 2013 · 76 citations · Full text

Autoimmune Disease, Allergy, Immunodeficiencies +11

Inspiratory muscle conditioning exercise and diaphragm gene therapy in Pompe disease: Clinical evidence of respiratory plasticity

Barbara K. Smith, A. Daniel Martin, Lee Ann Lawson et al. · Experimental Neurology · 2016 · 50 citations · Full text

Pompe Disease, Kinesiology, Pulmonary Care +9

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

Barry J. Byrne, Barbara K. Smith, Cathryn Mah et al. · Human Gene Therapy Clinical Development · 2014 · 45 citations