Recombinant adeno-associated virus gene therapy in light of Luxturna (and Zolgensma and Glybera): where are we, and how did we get here? AM Keeler, TR Flotte Annual review of virology 6 (1), 601-621, 2019 | 319 | 2019 |
Gene therapy 2017: progress and future directions AM Keeler, MK ElMallah, TR Flotte Clinical and translational science 10 (4), 242, 2017 | 190 | 2017 |
Lack of Cystic Fibrosis Transmembrane Conductance Regulator in CD3+ Lymphocytes Leads to Aberrant Cytokine Secretion and Hyperinflammatory Adaptive … C Mueller, SA Braag, A Keeler, C Hodges, M Drumm, TR Flotte American journal of respiratory cell and molecular biology 44 (6), 922-929, 2011 | 159 | 2011 |
Widespread central nervous system gene transfer and silencing after systemic delivery of novel AAV-AS vector SR Choudhury, AF Harris, DJ Cabral, AM Keeler, E Sapp, JS Ferreira, ... Molecular Therapy 24 (4), 726-735, 2016 | 151 | 2016 |
Death after high-dose rAAV9 gene therapy in a patient with Duchenne’s muscular dystrophy A Lek, B Wong, A Keeler, M Blackwood, K Ma, S Huang, K Sylvia, ... New England Journal of Medicine 389 (13), 1203-1210, 2023 | 134 | 2023 |
Systemic AAV9 gene transfer in adult GM1 gangliosidosis mice reduces lysosomal storage in CNS and extends lifespan CM Weismann, J Ferreira, AM Keeler, Q Su, L Qui, SA Shaffer, Z Xu, ... Human molecular genetics 24 (15), 4353-4364, 2015 | 113 | 2015 |
AAV gene therapy for Tay-Sachs disease TR Flotte, O Cataltepe, A Puri, AR Batista, R Moser, D McKenna-Yasek, ... Nature medicine 28 (2), 251-259, 2022 | 94 | 2022 |
CAR T-cell therapy: progress and prospects O Wilkins, AM Keeler, TR Flotte Human gene therapy methods 28 (2), 61-66, 2017 | 87 | 2017 |
CAR-T regulatory (CAR-Treg) cells: engineering and applications M Arjomandnejad, AL Kopec, AM Keeler Biomedicines 10 (2), 287, 2022 | 75 | 2022 |
Immunogenicity of recombinant adeno-associated virus (AAV) vectors for gene transfer M Arjomandnejad, I Dasgupta, TR Flotte, AM Keeler BioDrugs 37 (3), 311-329, 2023 | 72 | 2023 |
Systemic Delivery of AAVB1-GAA Clears Glycogen and Prolongs Survival in a Mouse Model of Pompe Disease AM Keeler, M Zieger, SH Todeasa, AL McCall, JC Gifford, S Birsak, ... Human Gene Therapy 30 (1), 57-68, 2019 | 45 | 2019 |
Cellular analysis of silencing the Huntington’s disease gene using AAV9 mediated delivery of artificial micro RNA into the striatum of Q140/Q140 mice AM Keeler, E Sapp, K Chase, E Sottosanti, E Danielson, E Pfister, ... Journal of Huntington's disease 5 (3), 239-248, 2016 | 41 | 2016 |
CRISPR/Cas-Dependent and Nuclease-Free In Vivo Therapeutic Gene Editing I Dasgupta, TR Flotte, AM Keeler Human gene therapy 32 (5-6), 275-293, 2021 | 39 | 2021 |
Long-term correction of very long-chain acyl-coA dehydrogenase deficiency in mice using AAV9 gene therapy AM Keeler, T Conlon, G Walter, H Zeng, SA Shaffer, F Dungtao, K Erger, ... Molecular Therapy 20 (6), 1131-1138, 2012 | 35 | 2012 |
Modulating immune responses to AAV by expanded polyclonal T-regs and capsid specific chimeric antigen receptor T-regulatory cells M Arjomandnejad, K Sylvia, M Blackwood, T Nixon, Q Tang, M Muhuri, ... Molecular Therapy Methods & Clinical Development 23, 490-506, 2021 | 31 | 2021 |
Novel combinatorial microRNA-binding sites in AAV vectors synergistically diminish antigen presentation and transgene immunity for efficient and stable transduction M Muhuri, W Zhan, Y Maeda, J Li, A Lotun, J Chen, K Sylvia, I Dasgupta, ... Frontiers in Immunology 12, 674242, 2021 | 29 | 2021 |
Intralingual and intrapleural AAV gene therapy prolongs survival in a SOD1 ALS mouse model AM Keeler, M Zieger, C Semple, L Pucci, A Veinbachs, RH Brown, ... Molecular Therapy-Methods & Clinical Development 17, 246-257, 2020 | 29 | 2020 |
Two-plasmid packaging system for recombinant adeno-associated virus Q Tang, AM Keeler, S Zhang, Q Su, Z Lyu, Y Cheng, G Gao, TR Flotte BioResearch Open Access 9 (1), 219-228, 2020 | 27 | 2020 |
Airway smooth muscle dysfunction in Pompe (Gaa−/−) mice AM Keeler, D Liu, M Zieger, L Xiong, J Salemi, K Bellvé, BJ Byrne, ... American Journal of Physiology-Lung Cellular and Molecular Physiology 312 (6 …, 2017 | 24 | 2017 |
Unexpected death of a Duchenne muscular dystrophy patient in an N-of-1 trial of rAAV9-delivered CRISPR-transactivator A Lek, B Wong, A Keeler, M Blackwood, K Ma, S Huang, K Sylvia, ... MedRxiv, 2023.05. 16.23289881, 2023 | 23 | 2023 |