Locatelli, Franco

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

Emapalumab in Children with Primary Hemophagocytic Lymphohistiocytosis

Locatelli, F., Jordan, M. B., Allen, C., Cesaro, S., Rizzari, C., Rao, A., Degar, B., Garrington, T. P., Sevilla, J., Putti, M.-C., et al. (2020) · New England Journal of Medicine

Autoimmune and Inflammatory Disorders ResearchImmunodeficiency and Autoimmune Disorders

Cited by 534

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

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

Hemopoietic stem cell transplantation in thalassemia: a report from the European Society for Blood and Bone Marrow Transplantation Hemoglobinopathy Registry, 2000–2010

Baronciani, D., Angelucci, E., Potschger, U., Gaziev, J., Yesilipek, A., Zecca, M., Orofino, M. G., Giardini, C., Al-Ahmari, A., Marktel, S., et al. (2016) · Bone Marrow Transplantation

Hematopoietic Stem Cell TransplantationHemoglobinopathies and Related Disorders

Cited by 241

Genomic subtyping and therapeutic targeting of acute erythroleukemia

Iacobucci, I., Wen, J., Meggendorfer, M., Choi, J. K., Shi, L., Pounds, S. B., Carmichael, C. L., Masih, K. E., Morris, S. M., Lindsley, R. C., et al. (2019) · Nature Genetics

Acute Myeloid Leukemia ResearchAcute Lymphoblastic Leukemia research

Cited by 155

Persistent B cell memory after SARS-CoV-2 vaccination is functional during breakthrough infections

Terreri, S., Piano Mortari, E., Vinci, M. R., Russo, C., Alteri, C., Albano, C., Colavita, F., Gramigna, G., Agrati, C., Linardos, G., et al. (2022) · Cell Host & Microbe

SARS-CoV-2 and COVID-19 ResearchImmune responses and vaccinations

Cited by 117

Eomesodermin controls a unique differentiation program in human IL‐10 and IFN‐γ coproducing regulatory T cells

Gruarin, P., Maglie, S., De Simone, M., Häringer, B., Vasco, C., Ranzani, V., Bosotti, R., Noddings, J. S., Larghi, P., Facciotti, F., et al. (2019) · European Journal of Immunology

T-cell and B-cell ImmunologyPsoriasis: Treatment and Pathogenesis

Cited by 92

Lenvatinib with etoposide plus ifosfamide in patients with refractory or relapsed osteosarcoma (ITCC-050): a multicentre, open-label, multicohort, phase 1/2 study

Gaspar, N., Venkatramani, R., Hecker-Nolting, S., Melcon, S. G., Locatelli, F., Bautista, F., Longhi, A., Lervat, C., Entz-Werle, N., Casanova, M., et al. (2021) · The Lancet Oncology

Sarcoma Diagnosis and TreatmentBone Tumor Diagnosis and Treatments

Cited by 89

Selective autophagy maintains centrosome integrity and accurate mitosis by turnover of centriolar satellites

Holdgaard, S. G., Cianfanelli, V., Pupo, E., Lambrughi, M., Lubas, M., Nielsen, J. C., Eibes, S., Maiani, E., Harder, L. M., Wesch, N., et al. (2019) · Nature Communications

Microtubule and mitosis dynamicsAutophagy in Disease and Therapy

Cited by 84

Phase I/II study of single-agent lenvatinib in children and adolescents with refractory or relapsed solid malignancies and young adults with osteosarcoma (ITCC-050)☆

Gaspar, N., Campbell-Hewson, Q., Gallego Melcon, S., Locatelli, F., Venkatramani, R., Hecker-Nolting, S., Gambart, M., Bautista, F., Thebaud, E., Aerts, I., et al. (2021) · ESMO Open

Sarcoma Diagnosis and TreatmentOvarian cancer diagnosis and treatment

Cited by 59

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

Lenvatinib Plus Ifosfamide and Etoposide in Children and Young Adults With Relapsed Osteosarcoma

Gaspar, N., Hung, G.-Y., Strauss, S. J., Campbell-Hewson, Q., Dela Cruz, F. S., Glade Bender, J. L., Koh, K.-N., Whittle, S. B., Chan, G. C.-F., Gerber, N. U., et al. (2024) · JAMA Oncology

Sarcoma Diagnosis and TreatmentBone Tumor Diagnosis and Treatments

Cited by 28

TFG binds LC3C to regulate ULK1 localization and autophagosome formation

Carinci, M., Testa, B., Bordi, M., Milletti, G., Bonora, M., Antonucci, L., Ferraina, C., Carro, M., Kumar, M., Ceglie, D., et al. (2021) · The EMBO Journal

Autophagy in Disease and TherapyCellular transport and secretion

Cited by 25

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