| 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 | 139 | 2016 |
| In vivo selection yields AAV-B1 capsid for central nervous system and muscle gene therapy SR Choudhury, Z Fitzpatrick, AF Harris, SA Maitland, JS Ferreira, Y Zhang, ... Molecular Therapy 24 (7), 1247-1257, 2016 | 131 | 2016 |
| CRISPR/Cas9-mediated targeted insertion of human F9 achieves therapeutic circulating protein levels in mice and non-human primates HR Huang, C Moroski-Erkul, P Bialek, C Wang, G Gong, S Hartfort, ... Mol. Ther 27 (4 Suppl 1), 7, 2019 | 17 | 2019 |
| Supra-therapeutic levels of transgene expression achieved in vivo by CRISPR/Cas9 mediated targeted gene insertion HR Huang, J Xie, C Moroski-Erkul, C Wang, G Gong, S Hartfort, R Sattler, ... HUMAN GENE THERAPY 29 (12), A26-A26, 2018 | | 2018 |
| Rapid Characterization of Transient Transfection Conditions for Scalable Production of Adeno-Associated Virus (AAV) Using NanoSight NS300 JT Panteli, L Li, S Povlich, A Harris, C Fulco, C Tipper, RK Clark, ... MOLECULAR THERAPY 25 (5), 328-328, 2017 | | 2017 |
| 263. A Novel Peptide-Grafted AAV Capsid Exhibits Enhanced CNS Transduction in Both Adult Mice and Cat, as Well as SOD1 Knockdown in Adult hSOD1 ALS Mice S Choudhury, L Stoica, AF Harris, D Cabral, HL Gray-Edwards, ... Molecular Therapy 23, S105, 2015 | | 2015 |
| In vivo selection yields AAV-B1 capsid for CNS and muscle gene therapy SR Choudhury, Z Fitzpatrick, AF Harris, SA Maitland | | 2014 |
