Lee Ann Lawson

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 · 155 citations · Full text

Medicine, Immunology, Pediatrics +17

B-cell depletion is protective against anti-AAV capsid immune response: a human subject case study

Manuela Corti, ME Elder, DJ Falk et al. · Molecular Therapy — Methods & Clinical Development · 2014 · 135 citations · Full text

Life Sciences, Immunology, Molecular Medicine +13

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 · 115 citations · Full text

Medicine, Hematology, Molecular Medicine +14

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 · 78 citations

Life Sciences, Biochemistry, Immunology +12

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

Medicine, Molecular Medicine, Physiology +17