Mapara, Markus

CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia

Frangoul, H., Altshuler, D., Cappellini, M. D., Chen, Y.-S., Domm, J., Eustace, B. K., Foell, J., de la Fuente, J., Grupp, S., Handgretinger, R., et al. (2021) · New England Journal of Medicine

Hemoglobinopathies and Related DisordersCRISPR and Genetic Engineering

Cited by 1,962

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

Exagamglogene Autotemcel for Transfusion-Dependent β-Thalassemia

Locatelli, F., Lang, P., Wall, D., Meisel, R., Corbacioglu, S., Li, A. M., de la Fuente, J., Shah, A. J., Carpenter, B., Kwiatkowski, J. L., et al. (2024) · New England Journal of Medicine

Hemoglobinopathies and Related DisordersVirus-based gene therapy research

Cited by 253

Safety and Efficacy of CTX001 in Patients with Transfusion-Dependent β-Thalassemia and Sickle Cell Disease: Early Results from the Climb THAL-111 and Climb SCD-121 Studies of Autologous CRISPR-CAS9-Modified CD34+ Hematopoietic Stem and Progenitor Cells

Frangoul, H., Bobruff, Y., Cappellini, M. D., Corbacioglu, S., Fernandez, C. M., de la Fuente, J., Grupp, S. A., Handgretinger, R., Ho, T. W., Imren, S., et al. (2020) · Blood

Hemoglobinopathies and Related DisordersVirus-based gene therapy research

Cited by 54

Gastrointestinal toxicity of high-dose melphalan in autologous hematopoietic stem cell transplantation: identification of risk factors and a benchmark for experimental therapies

Gordillo, C. A., Parmar, S., Blanco, M., Delille, E. M., Assal, A., Mapara, M., & Reshef, R. (2021) · Annals of Hematology

Hematopoietic Stem Cell TransplantationMultiple Myeloma Research and Treatments

Cited by 29

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