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Blood|January 8, 2003
AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia BCatherine S Manno, Amy J Chew, Sylvia Hutchison, et al.Molecular Therapy : the Journal of the American Society of Gene Therapy|December 3, 2009
Gene therapy for Leber's congenital amaurosis is safe and effective through 1.5 years after vector administrationFrancesca Simonelli, Albert M Maguire, Francesco Testa, et al.Cell Stem Cell|July 13, 2010
Patients beware: commercialized stem cell treatments on the webPatrick L Taylor, Roger A Barker, Karl G Blume, et al.Human Gene Therapy|March 23, 2013
Gene therapy for rare diseases: summary of a National Institutes of Health workshop, September 13, 2012Marina O'Reilly, Donald B Kohn, Jeffrey Bartlett, et al.Molecular Therapy : the Journal of the American Society of Gene Therapy|January 20, 2011
Long-term safety and efficacy following systemic administration of a self-complementary AAV vector encoding human FIX pseudotyped with serotype 5 and 8 capsid proteinsAmit C Nathwani, Cecilia Rosales, Jenny McIntosh, et al.Lancet (London, England)|July 5, 2016
Safety and durability of effect of contralateral-eye administration of AAV2 gene therapy in patients with childhood-onset blindness caused by RPE65 mutations: a follow-on phase 1 trialJean Bennett, Jennifer Wellman, Kathleen A Marshall, et al.Blood|February 22, 2013
Therapeutic levels of FVIII following a single peripheral vein administration of rAAV vector encoding a novel human factor VIII variantJenny McIntosh, Peter J Lenting, Cecilia Rosales, 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|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|December 7, 2017
Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX VariantLindsey A George, Spencer K Sullivan, Adam Giermasz, et al.Pageof 15