Margaret Humphries

Phase 2 Clinical Trial of a Recombinant Adeno-Associated Viral Vector Expressing α ₁ -Antitrypsin: Interim Results

Terence R. Flotte, Bruce C. Trapnell, Margaret Humphries et al. · Human Gene Therapy · 2011 · 322 citations · Full text

Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy

Mark Brantly, Jeffrey D. Chulay, Lili Wang et al. · Proceedings of the National Academy of Sciences · 2009 · 311 citations · Full text

Cell Therapy, In Vivo Gene Therapy, Transgene Expression +20

Phase I Trial of Intranasal and Endobronchial Administration of a Recombinant Adeno-Associated Virus Serotype 2 (rAAV2)-CFTR Vector in Adult Cystic Fibrosis Patients: A Two-Part Clinical Study

Terence R. Flotte, Pamela L. Zeitlin, Thomas C. Reynolds et al. · Human Gene Therapy · 2003 · 243 citations

Results at 2 Years after Gene Therapy for RPE65-Deficient Leber Congenital Amaurosis and Severe Early-Childhood–Onset Retinal Dystrophy

Richard G. Weleber, Mark E. Pennesi, David J. Wilson et al. · Ophthalmology · 2016 · 214 citations · Full text

Phase I Trial of Intramuscular Injection of a Recombinant Adeno-Associated Virus Serotype 2 <i>α</i> 1-Antitrypsin (AAT) Vector in AAT-Deficient Adults

Mark Brantly, L. Terry Spencer, Margaret Humphries et al. · Human Gene Therapy · 2006 · 174 citations