| Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial S Russell, J Bennett, JA Wellman, DC Chung, ZF Yu, A Tillman, J Wittes, ... The Lancet 390 (10097), 849-860, 2017 | 1628 | 2017 |
| Hemophilia B gene therapy with a high-specific-activity factor IX variant LA George, SK Sullivan, A Giermasz, JEJ Rasko, BJ Samelson-Jones, ... New England Journal of Medicine 377 (23), 2215-2227, 2017 | 715 | 2017 |
| Current clinical applications of in vivo gene therapy with AAVs JR Mendell, SA Al-Zaidy, LR Rodino-Klapac, K Goodspeed, SJ Gray, ... Molecular Therapy 29 (2), 464-488, 2021 | 487 | 2021 |
| Transmural heterogeneity of calcium activity and mechanical function in the canine left ventricle JM Cordeiro, L Greene, C Heilmann, D Antzelevitch, C Antzelevitch American Journal of Physiology-Heart and Circulatory Physiology 286 (4 …, 2004 | 250 | 2004 |
| Multiyear factor VIII expression after AAV gene transfer for hemophilia A LA George, PE Monahan, ME Eyster, SK Sullivan, MV Ragni, SE Croteau, ... New England Journal of Medicine 385 (21), 1961-1973, 2021 | 150 | 2021 |
| Long-term follow-up of the first in human intravascular delivery of AAV for gene transfer: AAV2-hFIX16 for severe hemophilia B LA George, MV Ragni, JEJ Rasko, LJ Raffini, BJ Samelson-Jones, ... Molecular Therapy 28 (9), 2073-2082, 2020 | 145 | 2020 |
| Beyond the platelet count: immature platelet fraction and thromboelastometry correlate with bleeding in patients with immune thrombocytopenia LA Greene, S Chen, C Seery, AM Imahiyerobo, JB Bussel British journal of haematology 166 (4), 592-600, 2014 | 106 | 2014 |
| Hemophilia gene therapy comes of age LA George Blood Advances 1 (26), 2591-2599, 2017 | 80 | 2017 |
| Deep vein thrombosis: thrombolysis in the pediatric population LA Greene, NA Goldenberg Seminars in interventional radiology 29 (01), 036-043, 2012 | 68 | 2012 |
| A phase 1/2 trial of investigational Spk-8011 in hemophilia a demonstrates durable expression and prevention of bleeds KA High, LA George, ME Eyster, SK Sullivan, MV Ragni, SE Croteau, ... Blood, The Journal of the American Society of Hematology 132 (Supplement 1 …, 2018 | 59 | 2018 |
| Novel factor VIII variants with a modified furin cleavage site improve the efficacy of gene therapy for hemophilia A GN Nguyen, LA George, JI Siner, RJ Davidson, CB Zander, XL Zheng, ... Journal of Thrombosis and Haemostasis 15 (1), 110-121, 2017 | 52 | 2017 |
| Adeno-associated virus gene therapy for hemophilia BJ Samelson-Jones, LA George Annual review of medicine 74, 231-247, 2023 | 51 | 2023 |
| Gene therapy for hemophilia: past, present and future LA George, PF Fogarty Seminars in hematology 53 (1), 46-54, 2016 | 50 | 2016 |
| Hyperactivity of factor IX Padua (R338L) depends on factor VIIIa cofactor activity BJ Samelson-Jones, JD Finn, LA George, RM Camire, VR Arruda JCI insight 4 (14), 2019 | 38 | 2019 |
| World federation of hemophilia gene therapy registry BA Konkle, D Coffin, GF Pierce, C Clark, L George, A Iorio, J Mahlangu, ... Haemophilia: the official journal of the World Federation of Hemophilia 26 …, 2020 | 34 | 2020 |
| The 1st WFH Gene Therapy Round Table: Understanding the landscape and challenges of gene therapy for haemophilia around the world GF Pierce, D Coffin, ... Haemophilia 25 (2), 189-194, 2019 | 33 | 2019 |
| Spk-8011: preliminary results from a phase 1/2 dose escalation trial of an investigational AAV-mediated gene therapy for hemophilia A LA George, MV Ragni, BJ Samelson-Jones, A Cuker, AR Runoski, G Cole, ... Blood 130, 604, 2017 | 33 | 2017 |
| Efficacy and safety in 15 hemophilia B patients treated with the AAV gene therapy vector fidanacogene elaparvovec and followed for at least 1 year LA George, SK Sullivan, JEJ Rasko, A Giermasz, BJ Samelson-Jones, ... Blood 134, 3347, 2019 | 30 | 2019 |
| Optimal management of autoimmune lymphoproliferative syndrome in children LA George, DT Teachey Pediatric Drugs 18, 261-272, 2016 | 26 | 2016 |
| Core data set on safety, efficacy, and durability of hemophilia gene therapy for a global registry: communication from the SSC of the ISTH B Konkle, G Pierce, D Coffin, M Naccache, RC Clark, L George, A Iorio, ... Journal of Thrombosis and Haemostasis 18 (11), 3074-3077, 2020 | 24 | 2020 |
