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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.
Orphanet Journal of Rare Diseases|August 2, 2018
Deep characterization of the anti-drug antibodies developed in Fabry disease patients, a prospective analysis from the French multicenter cohort FFABRYWladimir Mauhin, Olivier Lidove, Damien Amelin, et al.
Acta Neuropathologica Communications|October 30, 2019
Deep morphological analysis of muscle biopsies from type III glycogenesis (GSDIII), debranching enzyme deficiency, revealed stereotyped vacuolar myopathy and autophagy impairmentPascal Laforêt, Michio Inoue, Evelyne Goillot, et al.
Science Translational Medicine|December 1, 2017
Rescue of Pompe disease in mice by AAV-mediated liver delivery of secretable acid α-glucosidaseFrancesco Puzzo, Pasqualina Colella, Maria G Biferi, et al.
Science Advances|September 21, 2022
Semirational bioengineering of AAV vectors with increased potency and specificity for systemic gene therapy of muscle disordersJihad El Andari, Edith Renaud-Gabardos, Warut Tulalamba, 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.
Research and Practice in Thrombosis and Haemostasis|July 21, 2020
Illustrated State-of-the-Art Capsules of the ISTH 2020 CongressRobert Ariens, Cecilia Becattini, Markus Bender, et al.
Nature Communications|November 5, 2021
Hepatic expression of GAA results in enhanced enzyme bioavailability in mice and non-human primatesHelena Costa-Verdera, Fanny Collaud, Christopher R Riling, 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.
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