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Science Translational Medicine|January 24, 2014
Gene therapy prolongs survival and restores function in murine and canine models of myotubular myopathyMartin K Childers, Romain Joubert, Karine Poulard, et al.Molecular Therapy : the Journal of the American Society of Gene Therapy|August 25, 2011
Insertion sites in engrafted cells cluster within a limited repertoire of genomic areas after gammaretroviral vector gene therapyAnnette Deichmann, Martijn H Brugman, Cynthia C Bartholomae, et al.Molecular Therapy : the Journal of the American Society of Gene Therapy|February 27, 2017
Systemic AAV8-Mediated Gene Therapy Drives Whole-Body Correction of Myotubular Myopathy in DogsDavid L Mack, Karine Poulard, Melissa A Goddard, et al.Human Gene Therapy|February 6, 2015
Perspectives on best practices for gene therapy programsThomas R Cheever, Dale Berkley, Serge Braun, et al.Nature Medicine|December 8, 2009
Comprehensive genomic access to vector integration in clinical gene therapyRichard Gabriel, Ralph Eckenberg, Anna Paruzynski, et al.Nature Communications|April 1, 2025
Severe inflammation and lineage skewing are associated with poor engraftment of engineered hematopoietic stem cells in patients with sickle cell diseaseSteicy Sobrino, Laure Joseph, Elisa Magrin, et al.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.JAMA|April 22, 2015
Outcomes following gene therapy in patients with severe Wiskott-Aldrich syndromeSalima Hacein-Bey Abina, H Bobby Gaspar, Johanna Blondeau, et al.The Lancet. Neurology|November 17, 2023
Safety and efficacy of gene replacement therapy for X-linked myotubular myopathy (ASPIRO): a multinational, open-label, dose-escalation trialPerry B Shieh, Nancy L Kuntz, James J Dowling, et al.Pageof 11