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Nature Communications|July 26, 2017
Long-term microdystrophin gene therapy is effective in a canine model of Duchenne muscular dystrophyCaroline Le Guiner, Laurent Servais, Marie Montus, et al.Nature Medicine|February 14, 2006
Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune responseCatherine S Manno, Glenn F Pierce, Valder R Arruda, et al.The New England Journal of Medicine|April 29, 2008
Safety and efficacy of gene transfer for Leber's congenital amaurosisAlbert M Maguire, Francesca Simonelli, Eric A Pierce, et al.The New England Journal of Medicine|November 20, 2014
Long-term safety and efficacy of factor IX gene therapy in hemophilia BAmit C Nathwani, Ulreke M Reiss, Edward G D Tuddenham, et al.Lancet (London, England)|October 27, 2009
Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trialAlbert M Maguire, Katherine A High, Alberto Auricchio, et al.The New England Journal of Medicine|December 14, 2011
Adenovirus-associated virus vector-mediated gene transfer in hemophilia BAmit C Nathwani, Edward G D Tuddenham, Savita Rangarajan, et al.NEJM Evidence|February 6, 2024
Liver-Directed Adeno-Associated Virus-Mediated Gene Therapy for Mucopolysaccharidosis Type VINicola Brunetti-Pierri, Rita Ferla, Virginia Maria Ginocchio, et al.Science Translational Medicine|February 11, 2021
Engineering adeno-associated viral vectors to evade innate immune and inflammatory responsesYing Kai Chan, Sean K Wang, Colin J Chu, et al.Pageof 14