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

346 results

Filters applied: . Clear all
Results are displayed in a computed author sort order. The Results By Year timeline is not available.
Page 1
Gene therapy for immunodeficiency due to adenosine deaminase deficiency.
Aiuti A, Cattaneo F, Galimberti S, Benninghoff U, Cassani B, Callegaro L, Scaramuzza S, Andolfi G, Mirolo M, Brigida I, Tabucchi A, Carlucci F, Eibl M, Aker M, Slavin S, Al-Mousa H, Al Ghonaium A, Ferster A, Duppenthaler A, Notarangelo L, Wintergerst U, Buckley RH, Bregni M, Marktel S, Valsecchi MG, Rossi P, Ciceri F, Miniero R, Bordignon C, Roncarolo MG. Aiuti A, et al. Among authors: roncarolo mg. N Engl J Med. 2009 Jan 29;360(5):447-58. doi: 10.1056/NEJMoa0805817. N Engl J Med. 2009. PMID: 19179314 Free article. Clinical Trial.
Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning.
Aiuti A, Slavin S, Aker M, Ficara F, Deola S, Mortellaro A, Morecki S, Andolfi G, Tabucchi A, Carlucci F, Marinello E, Cattaneo F, Vai S, Servida P, Miniero R, Roncarolo MG, Bordignon C. Aiuti A, et al. Among authors: roncarolo mg. Science. 2002 Jun 28;296(5577):2410-3. doi: 10.1126/science.1070104. Science. 2002. PMID: 12089448 Clinical Trial.
Gene therapy for adenosine deaminase deficiency.
Aiuti A, Ficara F, Cattaneo F, Bordignon C, Roncarolo MG. Aiuti A, et al. Among authors: roncarolo mg. Curr Opin Allergy Clin Immunol. 2003 Dec;3(6):461-6. doi: 10.1097/00130832-200312000-00007. Curr Opin Allergy Clin Immunol. 2003. PMID: 14612670 Review.
Multilineage hematopoietic reconstitution without clonal selection in ADA-SCID patients treated with stem cell gene therapy.
Aiuti A, Cassani B, Andolfi G, Mirolo M, Biasco L, Recchia A, Urbinati F, Valacca C, Scaramuzza S, Aker M, Slavin S, Cazzola M, Sartori D, Ambrosi A, Di Serio C, Roncarolo MG, Mavilio F, Bordignon C. Aiuti A, et al. Among authors: roncarolo mg. J Clin Invest. 2007 Aug;117(8):2233-40. doi: 10.1172/JCI31666. J Clin Invest. 2007. PMID: 17671653 Free PMC article. Clinical Trial.
ADA-deficient SCID is associated with a specific microenvironment and bone phenotype characterized by RANKL/OPG imbalance and osteoblast insufficiency.
Sauer AV, Mrak E, Hernandez RJ, Zacchi E, Cavani F, Casiraghi M, Grunebaum E, Roifman CM, Cervi MC, Ambrosi A, Carlucci F, Roncarolo MG, Villa A, Rubinacci A, Aiuti A. Sauer AV, et al. Among authors: roncarolo mg. Blood. 2009 Oct 8;114(15):3216-26. doi: 10.1182/blood-2009-03-209221. Epub 2009 Jul 24. Blood. 2009. PMID: 19633200 Free article. Clinical Trial.
Integration of retroviral vectors induces minor changes in the transcriptional activity of T cells from ADA-SCID patients treated with gene therapy.
Cassani B, Montini E, Maruggi G, Ambrosi A, Mirolo M, Selleri S, Biral E, Frugnoli I, Hernandez-Trujillo V, Di Serio C, Roncarolo MG, Naldini L, Mavilio F, Aiuti A. Cassani B, et al. Among authors: roncarolo mg. Blood. 2009 Oct 22;114(17):3546-56. doi: 10.1182/blood-2009-02-202085. Epub 2009 Aug 3. Blood. 2009. PMID: 19652199 Free article. Clinical Trial.
Ten years of gene therapy for primary immune deficiencies.
Aiuti A, Roncarolo MG. Aiuti A, et al. Among authors: roncarolo mg. Hematology Am Soc Hematol Educ Program. 2009:682-9. doi: 10.1182/asheducation-2009.1.682. Hematology Am Soc Hematol Educ Program. 2009. PMID: 20008254 Review.
346 results