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JACC. Basic to Translational Science|July 30, 2025
Single Ascending-Dose Study of Selective ErbB4 Agonist JK07 in Heart Failure With Reduced Ejection FractionW H Wilson Tang, Johannes Steiner, Mahwash Kassi, et al.
Blood|March 26, 2010
Peripheral transvenular delivery of adeno-associated viral vectors to skeletal muscle as a novel therapy for hemophilia BValder R Arruda, Hansell H Stedman, Virginia Haurigot, et al.
Science Translational Medicine|July 19, 2013
Overcoming preexisting humoral immunity to AAV using capsid decoysFederico Mingozzi, Xavier M Anguela, Giulia Pavani, et al.
Ophthalmology|August 25, 2019
Efficacy, Safety, and Durability of Voretigene Neparvovec-rzyl in RPE65 Mutation-Associated Inherited Retinal Dystrophy: Results of Phase 1 and 3 TrialsAlbert M Maguire, Stephen Russell, Jennifer A Wellman, et al.
American Journal of Physiology. Heart and Circulatory Physiology|May 19, 2026
ERBB4 activation prevents microvascular dysfunction in a large animal model of hypertensive heart diseaseMichiel Rl Tubeeckx, Tijs Bringmans, Bo Goovaerts, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|January 23, 2008
Reversal of blindness in animal models of leber congenital amaurosis using optimized AAV2-mediated gene transferJeannette Bennicelli, John Fraser Wright, Andras Komaromy, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|June 28, 2012
Safe, long-term hepatic expression of anti-HCV shRNA in a nonhuman primate modelDavid A Suhy, Shih-Chu Kao, Tin Mao, et al.
The New England Journal of Medicine|November 17, 2021
Multiyear Factor VIII Expression after AAV Gene Transfer for Hemophilia ALindsey A George, Paul E Monahan, M Elaine Eyster, et al.
Science Translational Medicine|February 11, 2012
AAV2 gene therapy readministration in three adults with congenital blindnessJean Bennett, Manzar Ashtari, Jennifer Wellman, et al.
American Journal of Ophthalmology|October 1, 2018
The Natural History of Inherited Retinal Dystrophy Due to Biallelic Mutations in the RPE65 GeneDaniel C Chung, Mette Bertelsen, Birgit Lorenz, et al.
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