| Systemic γ-sarcoglycan AAV gene transfer results in dose-dependent correction of muscle deficits in the LGMD 2C/R5 mouse model YE Seo, SH Baine, AN Kempton, OC Rogers, S Lewis, K Adegboye, ... Molecular Therapy Methods & Clinical Development 28, 284-299, 2023 | 8 | 2023 |
| Evaluating pharmacology and efficacy of delandistrogene moxeparvovec in DMDmdx rats RA Potter, C Wier, S Baine, GC Olson, J Endres, A Kempton, L Clements, ... 27th International Annual Congress of the World Muscle Society (WMS …, 2022 | 1 | 2022 |
| Systemic dose-finding study with AAV-Mediated gamma-sarcoglycan gene therapy for treatment of muscle deficits in LGMD2C Mice Y Seo, A Kempton, O Rogers, S Baine, S Lewis, K Adegboye, A Haile, ... NEUROMUSCULAR DISORDERS 30, S87-S87, 2020 | 1 | 2020 |
| P17 Safety and efficacy of pre-treatment with imlifidase prior to AAV-based gene therapy in non-human primates with pre-existing anti-AAVrh74 antibodies R Potter, S Khan, J Snedeker, K Adegboye, A Haile, B Sayanjali, N Pukos, ... Neuromuscular Disorders 33, S101, 2023 | | 2023 |
| P23 Evaluation of safety parameters and dystrophin expression by sequential administration of exon-skipping and gene therapy in a DMDmdx mouse model R Potter, GC Olson, L Smith, J Greve, A Haile, C Wier, J Snedeker, ... Neuromuscular Disorders 33, S102-S103, 2023 | | 2023 |
| PK/PD modelling to inform clinical development of an adeno-associated virus gene transfer therapy for Duchenne muscular dystrophy L East, R Potter, J Snedeker, A Haile, C Wier, L Rodino-Klapac NEUROMUSCULAR DISORDERS 32, S127-S127, 2022 | | 2022 |
| P. 197 Evaluating pharmacology and efficacy of delandistrogene moxeparvovec in young and aged DMDMDX rats R Potter, C Wier, G Cooper-Olson, E Wheeler, E Anderbery, A Kempton, ... Neuromuscular Disorders 32, S127, 2022 | | 2022 |
| VP. 82 PK/PD modelling to inform clinical development of an adeno-associated virus gene transfer therapy for Duchenne muscular dystrophy L East, R Potter, J Snedeker, A Haile, C Wier, L Rodino-Klapac Neuromuscular Disorders 32, S127, 2022 | | 2022 |
| LIMB GIRDLE MUSCULAR DYSTROPHIES: P. 138 Systemic dose-finding study with AAV-Mediated gamma-sarcoglycan gene therapy for treatment of muscle deficits in LGMD2C Mice Y Seo, A Kempton, O Rogers, S Baine, S Lewis, K Adegboye, A Haile, ... Neuromuscular Disorders 30, S87, 2020 | | 2020 |
| Safety and efficacy of pre-treatment with imlifidase prior to AAV-based gene therapy in non-human primates with pre-existing anti-AAVrh74 antibodies RA Potter, S Khan, J Snedeker, K Adegboye, A Haile, N Pukos, ... | | |
Louise Rodino-KlapacCenter for Gene Therapy-Nationwide Children's HospitalVerified email at nationwidechildrens.org
