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Science Translational Medicine|April 9, 2010
Safety and efficacy of subretinal readministration of a viral vector in large animals to treat congenital blindnessDefne Amado, Federico Mingozzi, Daniel Hui, et al.
Ophthalmology|March 12, 2013
Three-year follow-up after unilateral subretinal delivery of adeno-associated virus in patients with Leber congenital Amaurosis type 2Francesco Testa, Albert M Maguire, Settimio Rossi, et al.
Molecular Therapy. Methods & Clinical Development|January 3, 2022
Preclinical assessment of an optimized AAV-FVIII vector in mice and non-human primates for the treatment of hemophilia ALiron Elkouby, Sean M Armour, Raffaella Toso, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|April 29, 2010
Safety of AAV factor IX peripheral transvenular gene delivery to muscle in hemophilia B dogsVirginia Haurigot, Federico Mingozzi, George Buchlis, et al.
The New England Journal of Medicine|April 16, 2025
Fidanacogene Elaparvovec for Hemophilia B - A Multiyear Follow-up StudyJohn E J Rasko, Benjamin J Samelson-Jones, Lindsey A George, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|February 9, 2017
Safety and Efficacy of Regional Intravenous (RI) Versus Intramuscular (IM) Delivery of rAAV1 and rAAV8 to Nonhuman Primate Skeletal MuscleAlice Toromanoff, Yan Chérel, Mickaël Guilbaud, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|May 8, 2008
Safety and efficacy of regional intravenous (r.i.) versus intramuscular (i.m.) delivery of rAAV1 and rAAV8 to nonhuman primate skeletal muscleAlice Toromanoff, Yan Chérel, Mickaël Guilbaud, et al.
Diabetes|February 5, 2013
Treatment of diabetes and long-term survival after insulin and glucokinase gene therapyDavid Callejas, Christopher J Mann, Eduard Ayuso, et al.
Blood|August 23, 2015
In vivo genome editing of the albumin locus as a platform for protein replacement therapyRajiv Sharma, Xavier M Anguela, Yannick Doyon, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|June 20, 2020
Long-Term Follow-Up of the First in Human Intravascular Delivery of AAV for Gene Transfer: AAV2-hFIX16 for Severe Hemophilia BLindsey A George, Margaret V Ragni, John E J Rasko, et al.
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