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

37 results

Filters applied: . Clear all
Results are displayed in a computed author sort order. The Results By Year timeline is not available.
Page 1
Long-term and real-world safety and efficacy of retroviral gene therapy for adenosine deaminase deficiency.
Migliavacca M, Barzaghi F, Fossati C, Rancoita PMV, Gabaldo M, Dionisio F, Giannelli S, Salerio FA, Ferrua F, Tucci F, Calbi V, Gallo V, Recupero S, Consiglieri G, Pajno R, Sambuco M, Priolo A, Ferri C, Garella V, Monti I, Silvani P, Darin S, Casiraghi M, Corti A, Zancan S, Levi M, Cesana D, Carlucci F, Pituch-Noworolska A, AbdElaziz D, Baumann U, Finocchi A, Cancrini C, Ladogana S, Meinhardt A, Meyts I, Montin D, Notarangelo LD, Porta F, Pasquet M, Speckmann C, Stepensky P, Tommasini A, Rabusin M, Karakas Z, Galicchio M, Leonardi L, Duse M, Guner SN, Di Serio C, Ciceri F, Bernardo ME, Aiuti A, Cicalese MP. Migliavacca M, et al. Among authors: calbi v. Nat Med. 2024 Feb;30(2):488-497. doi: 10.1038/s41591-023-02789-4. Epub 2024 Feb 14. Nat Med. 2024. PMID: 38355973
Use of Defibrotide to help prevent post-transplant endothelial injury in a genetically predisposed infant with metachromatic leukodystrophy undergoing hematopoietic stem cell gene therapy.
Calbi V, Fumagalli F, Consiglieri G, Penati R, Acquati S, Redaelli D, Attanasio V, Marcella F, Cicalese MP, Migliavacca M, Barzaghi F, Ferrua F, Assanelli A, Silvani P, Zoccolillo M, Chidini G, Chiesa R, Arora R, Ciotti F, Sarzana M, Antonioli G, Baldoli C, Morena F, Martino S, Ardissino GL, Sora MGN, Naldini L, Ciceri F, Aiuti A, Bernardo ME. Calbi V, et al. Bone Marrow Transplant. 2018 Jul;53(7):913-917. doi: 10.1038/s41409-017-0085-1. Epub 2018 Jan 29. Bone Marrow Transplant. 2018. PMID: 29379168 Free PMC article. No abstract available.
Successful Treatment With Ledipasvir/Sofosbuvir in an Infant With Severe Combined Immunodeficiency Caused by Adenosine Deaminase Deficiency With HCV Allowed Gene Therapy with Strimvelis.
Tucci F, Calbi V, Barzaghi F, Migliavacca M, Ferrua F, Bernardo ME, Canarutto D, Consiglieri G, Recupero S, Calzatini F, Gabaldo M, Lucano C, Casiraghi M, Darin S, Dionisio F, Marktel S, Cestone E, Finazzi R, Mieli-Vergani G, Boeri E, Appleby J, Abd Elaziz D, Ciceri F, Aiuti A, Cicalese MP. Tucci F, et al. Among authors: calbi v. Hepatology. 2018 Dec;68(6):2434-2437. doi: 10.1002/hep.30160. Epub 2018 Nov 13. Hepatology. 2018. PMID: 30014500 Free PMC article. No abstract available.
Intrabone hematopoietic stem cell gene therapy for adult and pediatric patients affected by transfusion-dependent ß-thalassemia.
Marktel S, Scaramuzza S, Cicalese MP, Giglio F, Galimberti S, Lidonnici MR, Calbi V, Assanelli A, Bernardo ME, Rossi C, Calabria A, Milani R, Gattillo S, Benedicenti F, Spinozzi G, Aprile A, Bergami A, Casiraghi M, Consiglieri G, Masera N, D'Angelo E, Mirra N, Origa R, Tartaglione I, Perrotta S, Winter R, Coppola M, Viarengo G, Santoleri L, Graziadei G, Gabaldo M, Valsecchi MG, Montini E, Naldini L, Cappellini MD, Ciceri F, Aiuti A, Ferrari G. Marktel S, et al. Among authors: calbi v. Nat Med. 2019 Feb;25(2):234-241. doi: 10.1038/s41591-018-0301-6. Epub 2019 Jan 21. Nat Med. 2019. PMID: 30664781 Clinical Trial.
Lentiviral haemopoietic stem/progenitor cell gene therapy for treatment of Wiskott-Aldrich syndrome: interim results of a non-randomised, open-label, phase 1/2 clinical study.
Ferrua F, Cicalese MP, Galimberti S, Giannelli S, Dionisio F, Barzaghi F, Migliavacca M, Bernardo ME, Calbi V, Assanelli AA, Facchini M, Fossati C, Albertazzi E, Scaramuzza S, Brigida I, Scala S, Basso-Ricci L, Pajno R, Casiraghi M, Canarutto D, Salerio FA, Albert MH, Bartoli A, Wolf HM, Fiori R, Silvani P, Gattillo S, Villa A, Biasco L, Dott C, Culme-Seymour EJ, van Rossem K, Atkinson G, Valsecchi MG, Roncarolo MG, Ciceri F, Naldini L, Aiuti A. Ferrua F, et al. Among authors: calbi v. Lancet Haematol. 2019 May;6(5):e239-e253. doi: 10.1016/S2352-3026(19)30021-3. Epub 2019 Apr 10. Lancet Haematol. 2019. PMID: 30981783 Free PMC article. Clinical Trial.
Bone marrow harvesting from paediatric patients undergoing haematopoietic stem cell gene therapy.
Tucci F, Frittoli M, Barzaghi F, Calbi V, Migliavacca M, Ferrua F, Fumagalli F, Lorioli L, Castagnaro L, Facchini M, Fossati C, Zancan S, Massariello P, Manfredini M, Consiglieri G, Canarutto D, Recupero S, Calzatini F, Casiraghi M, Darin S, Antonioli G, Miniero R, Fiori R, Silvani P, Zambelli M, Marktel S, Gattillo S, Milani R, Santoleri L, Ciceri F, Biffi A, Cicalese MP, Bernardo ME, Aiuti A. Tucci F, et al. Among authors: calbi v. Bone Marrow Transplant. 2019 Dec;54(12):1995-2003. doi: 10.1038/s41409-019-0573-6. Epub 2019 May 31. Bone Marrow Transplant. 2019. PMID: 31150018 Free PMC article.
Emapalumab treatment in an ADA-SCID patient with refractory hemophagocytic lymphohistiocytosis-related graft failure and disseminated bacillus Calmette-Guérin infection.
Tucci F, Gallo V, Barzaghi F, Ferrua F, Migliavacca M, Calbi V, Doglio M, Fratini ES, Karakas Z, Guner S, Zambelli M, Parisi C, Milani R, Gattillo S, Mazzi B, Oltolini C, Barbera M, Baldoli C, Cirillo DM, Asnaghi V, De Min C, Cicalese MP, Ciceri F, Aiuti A, Bernardo ME. Tucci F, et al. Among authors: calbi v. Haematologica. 2021 Feb 1;106(2):641-646. doi: 10.3324/haematol.2020.255620. Haematologica. 2021. PMID: 32817285 Free PMC article.
Mild SARS-CoV-2 Infection After Gene Therapy in a Child With Wiskott-Aldrich Syndrome: A Case Report.
Cenciarelli S, Calbi V, Barzaghi F, Bernardo ME, Oltolini C, Migliavacca M, Gallo V, Tucci F, Fraschetta F, Albertazzi E, Fratini ES, Consiglieri G, Giannelli S, Dionisio F, Sartirana C, Racca S, Camesasca C, Peretto G, Daverio R, Esposito A, De Cobelli F, Silvani P, Rabusin M, Cara A, Trabattoni D, Dispinseri S, Scarlatti G, Piemonti L, Lampasona V, Cicalese MP, Aiuti A, Ferrua F. Cenciarelli S, et al. Among authors: calbi v. Front Immunol. 2020 Nov 24;11:603428. doi: 10.3389/fimmu.2020.603428. eCollection 2020. Front Immunol. 2020. PMID: 33329599 Free PMC article.
37 results