Cavazzana, Marina

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

Cited by 696

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

A Modified γ-Retrovirus Vector for X-Linked Severe Combined Immunodeficiency

Hacein-Bey-Abina, S., Pai, S.-Y., Gaspar, H. B., Armant, M., Berry, C. C., Blanche, S., Bleesing, J., Blondeau, J., de Boer, H., Buckland, K. F., et al. (2014) · New England Journal of Medicine

Virus-based gene therapy researchImmunodeficiency and Autoimmune Disorders

Cited by 412

Induction of fetal hemoglobin synthesis by CRISPR/Cas9-mediated editing of the human β-globin locus

Antoniani, C., Meneghini, V., Lattanzi, A., Felix, T., Romano, O., Magrin, E., Weber, L., Pavani, G., El Hoss, S., Kurita, R., et al. (2018) · Blood

Hemoglobinopathies and Related DisordersCRISPR and Genetic Engineering

Cited by 194

Gene Therapy of the β-Hemoglobinopathies by Lentiviral Transfer of the β A(T87Q) - Globin Gene

Negre, O., Eggimann, A.-V., Beuzard, Y., Ribeil, J.-A., Bourget, P., Borwornpinyo, S., Hongeng, S., Hacein-Bey, S., Cavazzana, M., Leboulch, P., et al. (2016) · Human Gene Therapy

Virus-based gene therapy researchHemoglobinopathies and Related Disorders

Cited by 159

Editing a γ-globin repressor binding site restores fetal hemoglobin synthesis and corrects the sickle cell disease phenotype

Weber, L., Frati, G., Felix, T., Hardouin, G., Casini, A., Wollenschlaeger, C., Meneghini, V., Masson, C., De Cian, A., Chalumeau, A., et al. (2020) · Science Advances

Hemoglobinopathies and Related DisordersCRISPR and Genetic Engineering

Cited by 140

Preclinical Evaluation of Efficacy and Safety of an Improved Lentiviral Vector for the Treatment of β-Thalassemia and Sickle Cell Disease

Negre, O., Bartholomae, C., Beuzard, Y., Cavazzana, M., Christiansen, L., Courne, C., Deichmann, A., Denaro, M., Dreuzy, E., Finer, M., et al. (2014) · Current Gene Therapy

Virus-based gene therapy researchHemoglobinopathies and Related Disorders

Cited by 126

Gene therapy of hemoglobinopathies: progress and future challenges

Ikawa, Y., Miccio, A., Magrin, E., Kwiatkowski, J. L., Rivella, S., & Cavazzana, M. (2019) · Human Molecular Genetics

Virus-based gene therapy researchHemoglobinopathies and Related Disorders

Cited by 86

Hyperinflammation in patients with chronic granulomatous disease leads to impairment of hematopoietic stem cell functions

Weisser, M., Demel, U. M., Stein, S., Chen-Wichmann, L., Touzot, F., Santilli, G., Sujer, S., Brendel, C., Siler, U., Cavazzana, M., et al. (2016) · Journal of Allergy and Clinical Immunology

Immunodeficiency and Autoimmune DisordersHematopoietic Stem Cell Transplantation

Cited by 77

Sickle Cell Disease: From Genetics to Curative Approaches

Hardouin, G., Magrin, E., Corsia, A., Cavazzana, M., Miccio, A., & Semeraro, M. (2023) · Annual Review of Genomics and Human Genetics

Hemoglobinopathies and Related DisordersVirus-based gene therapy research

Cited by 40

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