Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation

Search Page

Filters

My NCBI Filters

Text availability

Article attribute

Article type

Publication date

Search Results

171 results

Filters applied: . Clear all
Results are displayed in a computed author sort order. The Results By Year timeline is not available.
Page 1
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.
Umbilical Cord Blood Transplantation for Fanconi Anemia With a Special Focus on Late Complications: a Study on Behalf of Eurocord and SAAWP-EBMT.
Rafii H, Volt F, Bierings M, Dalle JH, Ayas M, Rihani R, Faraci M, de Simone G, Sengeloev H, Passweg J, Cavazzana M, Costello R, Maertens J, Biffi A, Johansson JE, Montoro J, Guepin GR, Diaz MA, Sirvent A, Kenzey C, Rivera Franco MM, Cappelli B, Scigliuolo GM, Rocha V, Ruggeri A, Risitano A, De Latour RP, Gluckman E. Rafii H, et al. Among authors: cavazzana m. Transplant Cell Ther. 2024 May;30(5):532.e1-532.e16. doi: 10.1016/j.jtct.2024.02.024. Epub 2024 Mar 5. Transplant Cell Ther. 2024. PMID: 38452872 Free article.
171 results