| A long-term study of AAV gene therapy in dogs with hemophilia A identifies clonal expansions of transduced liver cells GN Nguyen, JK Everett, S Kafle, AM Roche, HE Raymond, J Leiby, ... Nature biotechnology 39 (1), 47-55, 2021 | 300 | 2021 |
| Long-term AAV-mediated factor VIII expression in nine hemophilia A dogs: a 10 year follow-up analysis on durability, safety and vector integration GN Nguyen, JK Everett, H Raymond, S Kafle, EP Merricks, HH Kazazian, ... Blood 134, 611, 2019 | 23 | 2019 |
| Wood, 513 C., Assenmacher, CA, Merricks, EP, Long, CT, et al.(2021). A long-term study of 514 AAV gene therapy in dogs with hemophilia A identifies clonal expansions of … GN Nguyen, JK Everett, S Kafle, AM Roche, HE Raymond, J Leiby Nat. Biotechnol 39, 47-55, 0 | 5 | |
| Altered cleavage of human factor VIII at the B-domain and acidic region 3 interface enhances expression after gene therapy in hemophilia A mice GN Nguyen, JR Lindgren, MC Seleme, S Kafle, CB Zander, XL Zheng, ... Journal of Thrombosis and Haemostasis 21 (8), 2101-2113, 2023 | 3 | 2023 |
| Multiple and consecutive genome editing using i-GONAD and breeding enrichment facilitates the production of genetically modified mice CR Melo-Silva, CJ Knudson, L Tang, S Kafle, LE Springer, J Choi, ... Cells 12 (9), 1343, 2023 | 2 | 2023 |
| Resistance to poxvirus lethality does not require the necroptosis proteins RIPK3 or MLKL B Montoya, CJ Knudson, CR Melo-Silva, L Tang, S Kafle, LJ Sigal Journal of Virology 97 (2), e01945-22, 2023 | 1 | 2023 |
| AAV integration analysis after long term follow up in hemophilia a dogs reveals the genetic consequences of AAV-mediated gene correction JK Everett, GN Nguyen, H Raymond, A Roche, S Kafle, C Wood, J Leiby, ... MOLECULAR THERAPY 28 (4), 409-409, 2020 | 1 | 2020 |
| mRNA-LNP vaccine-induced CD8+ T-cells protect mice from lethal SARS-CoV-2 infection in the absence of specific antibodies. B Montoya, CR Melo-Silva, L Tang, S Kafle, P Lidskiy, C Bajusz, ... Molecular Therapy, 2024 | | 2024 |
Christian WoodPerelman School of MedicineVerified email at pennmedicine.upenn.edu
