Soni, Sandeep

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

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Gene therapy in a patient with sickle cell disease

Ribeil, J.-A., Hacein-Bey-Abina, S., Payen, E., Magnani, A., Semeraro, M., Magrin, E., Caccavelli, L., Neven, B., Bourget, P., El Nemer, W., et al. (2017) · New England Journal of Medicine

Virus-based gene therapy researchHemoglobinopathies and Related Disorders

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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

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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

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