Mark Brantly

Efficacy and Safety of Sirolimus in Lymphangioleiomyomatosis

Francis X. McCormack, Yoshikazu Inoue, Joel Moss et al. · New England Journal of Medicine · 2011 · 1.1K citations · Full text

Pharmacy, Pharmacology, Pharmacotherapy +13

Hepatocytes corrected by gene therapy are selected in vivo in a murine model of hereditary tyrosinaemia type I

Ken Overturf, Muhsen Al-Dhalimy, Robert M. Tanguay et al. · Nature Genetics · 1996 · 555 citations

Life Sciences, Genetics, Molecular Biology +13

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 · 327 citations · Full text

Pharmacy, Pharmaceutical Biotechnology, Gene Delivery +12

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 · 320 citations · Full text

Medicine, Immunology, Molecular Medicine +15

Genetic Defects and Clinical Characteristics of Patients with a Form of Oculocutaneous Albinism (Hermansky–Pudlak Syndrome)

W. A. Gahl, Mark Brantly, Muriel I. Kaiser‐Kupfer et al. · New England Journal of Medicine · 1998 · 309 citations · Full text

Life Sciences, Genetics, Molecular Genetics +15