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Haematopoietic gene therapy of non-conditioned patients with Fanconi anaemia-A: results from open-label phase 1/2 (FANCOLEN-1) and long-term clinical trials.
Río P, Zubicaray J, Navarro S, Gálvez E, Sánchez-Domínguez R, Nicoletti E, Sebastián E, Rothe M, Pujol R, Bogliolo M, John-Neek P, Bastone AL, Schambach A, Wang W, Schmidt M, Larcher L, Segovia JC, Yáñez RM, Alberquilla O, Díez B, Fernández-García M, García-García L, Ramírez M, Galy A, Lefrere F, Cavazzana M, Leblanc T, García de Andoin N, López-Almaraz R, Catalá A, Barquinero J, Rodríguez-Perales S, Rao G, Surrallés J, Soulier J, Díaz-de-Heredia C, Schwartz JD, Sevilla J, Bueren JA; FANCOLEN-1 gene therapy investigators. Río P, et al. Among authors: cavazzana m. Lancet. 2025 Dec 21;404(10471):2584-2592. doi: 10.1016/S0140-6736(24)01880-4. Epub 2024 Dec 3. Lancet. 2025. PMID: 39642902 Clinical Trial.
Treating Sickle Cell Disease: Gene Therapy Approaches.
Cavazzana M, Corsia A, Brusson M, Miccio A, Semeraro M. Cavazzana M, et al. Annu Rev Pharmacol Toxicol. 2024 Sep 11. doi: 10.1146/annurev-pharmtox-022124-022000. Online ahead of print. Annu Rev Pharmacol Toxicol. 2024. PMID: 39259977 Review.
RAC2 gain-of-function variants causing inborn error of immunity drive NLRP3 inflammasome activation.
Doye A, Chaintreuil P, Lagresle-Peyrou C, Batistic L, Marion V, Munro P, Loubatier C, Chirara R, Sorel N, Bessot B, Bronnec P, Contenti J, Courjon J, Giordanengo V, Jacquel A, Barbry P, Couralet M, Aladjidi N, Fischer A, Cavazzana M, Mallebranche C, Visvikis O, Kracker S, Moshous D, Verhoeyen E, Boyer L. Doye A, et al. Among authors: cavazzana m. J Exp Med. 2024 Oct 7;221(10):e20231562. doi: 10.1084/jem.20231562. Epub 2024 Aug 30. J Exp Med. 2024. PMID: 39212656
Restoration of T and B Cell Differentiation after RAG1 Gene Transfer in Human RAG1 Defective Hematopoietic Stem Cells.
Sorel N, Díaz-Pascual F, Bessot B, Sadek H, Mollet C, Chouteau M, Zahn M, Gil-Farina I, Tajer P, van Eggermond M, Berghuis D, Lankester AC, André I, Gabriel R, Cavazzana M, Pike-Overzet K, Staal FJT, Lagresle-Peyrou C. Sorel N, et al. Among authors: cavazzana m. Biomedicines. 2024 Jul 5;12(7):1495. doi: 10.3390/biomedicines12071495. Biomedicines. 2024. PMID: 39062069 Free PMC article.
Non-viral DNA delivery and TALEN editing correct the sickle cell mutation in hematopoietic stem cells.
Moiani A, Letort G, Lizot S, Chalumeau A, Foray C, Felix T, Le Clerre D, Temburni-Blake S, Hong P, Leduc S, Pinard N, Marechal A, Seclen E, Boyne A, Mayer L, Hong R, Pulicani S, Galetto R, Gouble A, Cavazzana M, Juillerat A, Miccio A, Duclert A, Duchateau P, Valton J. Moiani A, et al. Among authors: cavazzana m. Nat Commun. 2024 Jun 11;15(1):4965. doi: 10.1038/s41467-024-49353-3. Nat Commun. 2024. PMID: 38862518 Free PMC article.
A case of T-cell acute lymphoblastic leukemia in retroviral gene therapy for ADA-SCID.
Cesana D, Cicalese MP, Calabria A, Merli P, Caruso R, Volpin M, Rudilosso L, Migliavacca M, Barzaghi F, Fossati C, Gazzo F, Pizzi S, Ciolfi A, Bruselles A, Tucci F, Spinozzi G, Pais G, Benedicenti F, Barcella M, Merelli I, Gallina P, Giannelli S, Dionisio F, Scala S, Casiraghi M, Strocchio L, Vinti L, Pacillo L, Draghi E, Cesana M, Riccardo S, Colantuono C, Six E, Cavazzana M, Carlucci F, Schmidt M, Cancrini C, Ciceri F, Vago L, Cacchiarelli D, Gentner B, Naldini L, Tartaglia M, Montini E, Locatelli F, Aiuti A. Cesana D, et al. Among authors: cavazzana m. Nat Commun. 2024 Apr 30;15(1):3662. doi: 10.1038/s41467-024-47866-5. Nat Commun. 2024. PMID: 38688902 Free PMC article.
Exagamglogene Autotemcel for Severe Sickle Cell Disease.
Frangoul H, Locatelli F, Sharma A, Bhatia M, Mapara M, Molinari L, Wall D, Liem RI, Telfer P, Shah AJ, Cavazzana M, Corbacioglu S, Rondelli D, Meisel R, Dedeken L, Lobitz S, de Montalembert M, Steinberg MH, Walters MC, Eckrich MJ, Imren S, Bower L, Simard C, Zhou W, Xuan F, Morrow PK, Hobbs WE, Grupp SA; CLIMB SCD-121 Study Group. Frangoul H, et al. Among authors: cavazzana m. N Engl J Med. 2024 May 9;390(18):1649-1662. doi: 10.1056/NEJMoa2309676. Epub 2024 Apr 24. N Engl J Med. 2024. PMID: 38661449 Clinical Trial.
Cellular Therapy with Engineered T Cells, Efficacy, and Side Effects: Gene Editing/Gene Therapy.
Bonini C, Cavazzana M, Ciceri F, Fehse B, Hudecek M. Bonini C, et al. Among authors: cavazzana m. 2024 Apr 11. In: Sureda A, Corbacioglu S, Greco R, Kröger N, Carreras E, editors. The EBMT Handbook: Hematopoietic Cell Transplantation and Cellular Therapies [Internet]. 8th edition. Cham (CH): Springer; 2024. Chapter 60. 2024 Apr 11. In: Sureda A, Corbacioglu S, Greco R, Kröger N, Carreras E, editors. The EBMT Handbook: Hematopoietic Cell Transplantation and Cellular Therapies [Internet]. 8th edition. Cham (CH): Springer; 2024. Chapter 60. PMID: 39437122 Free Books & Documents. Review.
173 results