Brian Cleaver

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

RNA aggregates harness the danger response for potent cancer immunotherapy

Héctor R. Méndez‐Gómez, Anna DeVries, Christina A. Von Roemeling et al. · Cell · 2024 · 131 citations · Full text

Life Sciences, Molecular Biology, Oncology +13

Preclinical Potency and Safety Studies of an AAV2-Mediated Gene Therapy Vector for the Treatment of <i>MERTK</i> Associated Retinitis Pigmentosa

Thomas J. Conlon, Wen‐Tao Deng, Kirsten Erger et al. · Human Gene Therapy Clinical Development · 2013 · 95 citations · Full text

Medicine, Molecular Biology, Ophthalmology +15

Evaluation of Readministration of a Recombinant Adeno-Associated Virus Vector Expressing Acid Alpha-Glucosidase in Pompe Disease: Preclinical to Clinical Planning

Manuela Corti, Brian Cleaver, Nathalie Clément et al. · Human Gene Therapy Clinical Development · 2015 · 91 citations · Full text

Medicine, Molecular Biology, Molecular Genetics +14

A simplified purification protocol for recombinant adeno-associated virus vectors

Mark Potter, Bridget Lins, Mario Mietzsch et al. · Molecular Therapy — Methods & Clinical Development · 2014 · 75 citations · Full text

Life Sciences, Biotechnology, Molecular Biology +14