Walters, Mark C.

Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia

Thompson, A. A., Walters, M. C., Kwiatkowski, J., Rasko, J. E. J., Ribeil, J.-A., Hongeng, S., Magrin, E., Schiller, G. J., Payen, E., Semeraro, M., et al. (2018) · New England Journal of Medicine

Virus-based gene therapy researchHemoglobinopathies and Related Disorders

Cited by 684

Exagamglogene Autotemcel for Severe Sickle Cell Disease

Frangoul, H., Locatelli, F., Sharma, A., Bhatia, M., Mapara, M., Molinari, L., Wall, D., Liem, R. I., Telfer, P., Shah, A. J., et al. (2024) · New England Journal of Medicine

Hemoglobinopathies and Related DisordersVirus-based gene therapy research

Cited by 493

Selection-free genome editing of the sickle mutation in human adult hematopoietic stem/progenitor cells

DeWitt, M. A., Magis, W., Bray, N. L., Wang, T., Berman, J. R., Urbinati, F., Heo, S.-J., Mitros, T., Muñoz, D. P., Boffelli, D., et al. (2016) · Science Translational Medicine

CRISPR and Genetic EngineeringHemoglobinopathies and Related Disorders

Cited by 477

Outcome of patients with hemoglobinopathies given either cord blood or bone marrow transplantation from an HLA-identical sibling

Locatelli, F., Kabbara, N., Ruggeri, A., Ghavamzadeh, A., Roberts, I., Li, C. K., Bernaudin, F., Vermylen, C., Dalle, J.-H., Stein, J., et al. (2013) · Blood

Hematopoietic Stem Cell TransplantationHemoglobinopathies and Related Disorders

Cited by 244

Myelodysplastic syndrome unrelated to lentiviral vector in a patient treated with gene therapy for sickle cell disease

Hsieh, M. M., Bonner, M., Pierciey, F. J., Uchida, N., Rottman, J., Demopoulos, L., Schmidt, M., Kanter, J., Walters, M. C., Thompson, A. A., et al. (2020) · Blood Advances

Virus-based gene therapy researchHemoglobinopathies and Related Disorders

Cited by 152

Resolution of Sickle Cell Disease Manifestations in Patients Treated with Lentiglobin Gene Therapy: Updated Results from the Phase 1/2 Hgb-206 Group C Study

Kanter, J., Tisdale, J. F., Mapara, M. Y., Kwiatkowski, J. L., Krishnamurti, L., Schmidt, M., Miller, A. L., Pierciey, F. J., Huang, W., Ribeil, J.-A., et al. (2019) · Blood

Hemoglobinopathies and Related DisordersVirus-based gene therapy research

Cited by 21

Efficacy, Safety, and Health-Related Quality of Life (HRQOL) in Patients with Sickle Cell Disease (SCD) Who Have Received Lovotibeglogene Autotemcel (Lovo-cel) Gene Therapy: Up to 60 Months of Follow-up

Kanter, J., Thompson, A. A., Kwiatkowski, J. L., Parikh, S., Mapara, M., Rifkin-Zenenberg, S., Aygun, B., Kasow, K. A., Gupta, A. O., Zhang, L., et al. (2023) · Blood

Hemoglobinopathies and Related DisordersNeurogenetic and Muscular Disorders Research

Cited by 15

Sustained Efficacy, Safety, and Improved Quality of Life in Adult and Pediatric Patients with Transfusion-Dependent β-Thalassemia up to 9 Years Post Treatment with Betibeglogene Autotemcel (Beti-cel)

Thompson, A. A., Olson, T. S., Walters, M. C., Porter, J. B., Schneiderman, J., Hongeng, S., Kulozik, A., Cavazzana, M., Sauer, M. G., Thrasher, A. J., et al. (2023) · Blood

Virus-based gene therapy researchHemoglobinopathies and Related Disorders

Cited by 6