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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.
Ovarian tissue cryopreservation for fertility preservation before hematopoietic stem cell transplantation in patients with sickle cell disease: safety, ovarian function follow-up, and results of ovarian tissue transplantation.
Missontsa MM, Bernaudin F, Fortin A, Dhédin N, Pondarré C, Yakouben K, Neven B, Castelle M, Cavazzana M, Lezeau H, Peycelon M, Paye-Jaouen A, Sroussi J, Diesch-Furlanetto T, Barraud-Lange V, Sarnacki S, Fahd M, Marchand I, Delcour C, Vexiau D, Arlet JB, Kamdem A, Arnaud C, Dalle JH, Poirot C. Missontsa MM, et al. Among authors: cavazzana m. J Assist Reprod Genet. 2024 Apr;41(4):1027-1034. doi: 10.1007/s10815-024-03054-4. Epub 2024 Feb 15. J Assist Reprod Genet. 2024. PMID: 38358434
Rescuing the cytolytic function of APDS1 patient T cells via TALEN-mediated PIK3CD gene correction.
Poggi L, Chentout L, Lizot S, Boyne A, Juillerat A, Moiani A, Luka M, Carbone F, Ménager M, Cavazzana M, Duchateau P, Valton J, Kracker S. Poggi L, et al. Among authors: cavazzana m. Mol Ther Methods Clin Dev. 2023 Oct 10;31:101133. doi: 10.1016/j.omtm.2023.101133. eCollection 2023 Dec 14. Mol Ther Methods Clin Dev. 2023. PMID: 38152700 Free PMC article.
Sickle Cell Disease: From Genetics to Curative Approaches.
Hardouin G, Magrin E, Corsia A, Cavazzana M, Miccio A, Semeraro M. Hardouin G, et al. Among authors: cavazzana m. Annu Rev Genomics Hum Genet. 2023 Aug 25;24:255-275. doi: 10.1146/annurev-genom-120122-081037. Annu Rev Genomics Hum Genet. 2023. PMID: 37624668 Free article. Review.
Activated phosphoinositide 3-kinase δ syndrome: Update from the ESID Registry and comparison with other autoimmune-lymphoproliferative inborn errors of immunity.
Maccari ME, Wolkewitz M, Schwab C, Lorenzini T, Leiding JW, Aladjdi N, Abolhassani H, Abou-Chahla W, Aiuti A, Azarnoush S, Baris S, Barlogis V, Barzaghi F, Baumann U, Bloomfield M, Bohynikova N, Bodet D, Boutboul D, Bucciol G, Buckland MS, Burns SO, Cancrini C, Cathébras P, Cavazzana M, Cheminant M, Chinello M, Ciznar P, Coulter TI, D'Aveni M, Ekwall O, Eric Z, Eren E, Fasth A, Frange P, Fournier B, Garcia-Prat M, Gardembas M, Geier C, Ghosh S, Goda V, Hammarström L, Hauck F, Heeg M, Heropolitanska-Pliszka E, Hilfanova A, Jolles S, Karakoc-Aydiner E, Kindle GR, Kiykim A, Klemann C, Koletsi P, Koltan S, Kondratenko I, Körholz J, Krüger R, Jeziorski E, Levy R, Le Guenno G, Lefevre G, Lougaris V, Marzollo A, Mahlaoui N, Malphettes M, Meinhardt A, Merlin E, Meyts I, Milota T, Moreira F, Moshous D, Mukhina A, Neth O, Neubert J, Neven B, Nieters A, Nove-Josserand R, Oksenhendler E, Ozen A, Olbrich P, Perlat A, Pac M, Schmid JP, Pacillo L, Parra-Martinez A, Paschenko O, Pellier I, Sefer AP, Plebani A, Plantaz D, Prader S, Raffray L, Ritterbusch H, Riviere JG, Rivalta B, Rusch S, Sakovich I, Savic S, Scheible R, Schleinitz N, Schuetz C, Schulz A, Sediva A, Semeraro M, Sharapova SO,… See abstract for full author list ➔ Maccari ME, et al. Among authors: cavazzana m. J Allergy Clin Immunol. 2023 Oct;152(4):984-996.e10. doi: 10.1016/j.jaci.2023.06.015. Epub 2023 Jun 28. J Allergy Clin Immunol. 2023. PMID: 37390899
Novel lentiviral vectors for gene therapy of sickle cell disease combining gene addition and gene silencing strategies.
Brusson M, Chalumeau A, Martinucci P, Romano O, Felix T, Poletti V, Scaramuzza S, Ramadier S, Masson C, Ferrari G, Mavilio F, Cavazzana M, Amendola M, Miccio A. Brusson M, et al. Among authors: cavazzana m. Mol Ther Nucleic Acids. 2023 Mar 22;32:229-246. doi: 10.1016/j.omtn.2023.03.012. eCollection 2023 Jun 13. Mol Ther Nucleic Acids. 2023. PMID: 37090420 Free PMC article.
166 results