Punam Malik, MD
Affiliations: | Cincinatti Children's Hospital |
Area:
Stem cell gene therapyGoogle:
"Punam Malik"Parents
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Publications
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| Shrestha A, Pillis DM, Felker S, et al. (2025) Preclinical efficacy of a modified gamma-globin lentivirus gene therapy in Berkeley sickle cell anemia mice and human xenograft models. Molecular Therapy. Methods & Clinical Development. 33: 101439 |
| Shadid M, Shrestha A, Malik P. (2024) Preclinical safety assessment of modified gamma globin lentiviral vector-mediated autologous hematopoietic stem cell gene therapy for hemoglobinopathies. Plos One. 19: e0306719 |
| McDonald CL, Qasba P, Anderson DG, et al. (2023) Future Directions and Resource Needs for National Heart, Lung, and Blood Institute (NHLBI) Gene Therapy Research: A Report of an NHLBI Workshop. Human Gene Therapy. 34: 83-89 |
| Smith RH, Bloomer H, Fink D, et al. (2022) Preclinical Evaluation of Foamy Virus Vector-Mediated Gene Addition in Human Hematopoietic Stem/Progenitor Cells for Correction of LAD-1. Human Gene Therapy |
| Cannon P, Asokan A, Czechowicz A, et al. (2021) Safe and Effective In Vivo Targeting and Gene Editing in Hematopoietic Stem Cells: Strategies for Accelerating Development National Institutes of Health/Bill & Melinda Gates Foundation Expert Scientific Roundtable Webinar Meeting. Human Gene Therapy |
| Farrell AT, Panepinto J, Desai AA, et al. (2019) End points for sickle cell disease clinical trials: renal and cardiopulmonary, cure, and low-resource settings. Blood Advances. 3: 4002-4020 |
| Hetzel M, Lopez-Rodriguez E, Mucci A, et al. (2019) Effective hematopoietic stem cell-based gene therapy in a murine model of hereditary pulmonary alveolar proteinosis. Haematologica |
| Jayavaradhan R, Pillis DM, Goodman M, et al. (2019) CRISPR-Cas9 fusion to dominant-negative 53BP1 enhances HDR and inhibits NHEJ specifically at Cas9 target sites. Nature Communications. 10: 2866 |
| Nasimuzzaman M, Arumugam PI, Mullins ES, et al. (2019) Elimination of the fibrinogen integrin αβ-binding motif improves renal pathology in mice with sickle cell anemia. Blood Advances. 3: 1519-1532 |
| Jayavaradhan R, Malik P. (2018) Genetic Therapies for Sickle Cell Disease. Pediatric Clinics of North America. 65: 465-480 |