Free Full-Text Articles
The article links below provide free access to the full-text versions of the most important journal articles of interest in the field of ADA-SCID, as selected by our Editorial Board.
Please click on the relevant article link to access the free full-text download for each article.
Maddalena Migliavacca, Andrea Assanelli, Maurilio Ponzoni, Roberta Pajno, Federica Barzaghi, Fabio Giglio, Francesca Ferrua, Marta Frittoli, Immacolata Brigida, Francesca Dionisio, Roberto Nicoletti, Miriam Casiraghi, Maria Grazia Roncarolo, Claudio Doglioni, Jacopo Peccatori, Fabio Ciceri, Maria Pia Cicalese, Alessandro Aiuti
Front Immunol. 2018; 9:113.
Maria Pia Cicalese, Francesca Ferrua, Laura Castagnaro, Katie Rolfe, Erika De Boever, Rickey R. Reinhardt, Jonathan Appleby, Maria Grazia Roncarolo, and Alessandro Aiuti
Mol Ther. 2018; 26(3):917-931
Single Cell-Based Vector Tracing in Patients with ADA-SCID Treated with Stem Cell Gene Therapy
Yuka Igarashi, Toru Uchiyama, Tomoko Minegishi, Sirirat Takahashi, Nobuyuki Watanabe, Toshinao Kawai, Masafumi Yamada, Tadashi Ariga and Masafumi Onodera
Mol Ther Methods Clin Dev. 2017; 6: 8-16
Twenty-Five Years of Gene Therapy for ADA-SCID: From Bubble Babies to an Approved Drug
Ferrua F, Aiuti A
Hum Gene Ther. 2017; 28(11):972-981.
Long-Term Outcome of Adenosine Deaminase-Deficient Patients—a Single-Center Experience
Ori Scott, Vy Kim, Brenda Reid, Anne Pham-Huy, Adelle Atkinson, Alessandro Aiuti, Eyal Grunebaum
J Clin Immunol. 2017; 37: 582-591
Kathryn L. Bradford, Federico A. Moretti, Denise A. Carbonaro-Sarracino, Hubert B. Gaspar, Donald B. Kohn
J Clin Immunol. 2017; 37: 626-37
Nicola Carriglio, Jan Klapwijk, Raisa Jofra Hernandez, Michela Vezzoli, Franck Chanut, Rhiannon Lowe, Draghici Elena, Melanie Nord, Paola Albertini, Patrizia Cristofori, Jane Richards, Hazel Staton, Jonathan Appleby, Alessandro Aiuti, Aisha V Sauer
Human Gene Ther Clin Dev. 2017; 28:17-27
Aaron R. Cooper, Georgia R. Lill, Kit Shaw, Denise A. Carbonaro-Sarracino, Alejandra Davila, Robert Sokolic, Fabio Candotti, Matteo Pellegrini, and Donald B. Kohn
Blood. 2017; 129:2624-35.
Aisha V Sauer, Raisa Jofra Hernandez, Francesca Fumagalli, Veronica Bianchi, Pietro L Poliani, Chiara Dallatomasina, Elisa Riboni, Letterio S Politi, Antonella Tabucchi, Filippo Carlucci, Miriam Casiraghi, Nicola Carriglio, Manuela Cominelli, Carlo Alberto Forcellini, Federica Barzaghi, Francesca Ferrua, Fabio Minicucci, Stefania Medaglini, Letizia Leocani, Giancarlo la Marca, Lucia D Notarangelo, Chiara Azzari, Giancarlo Comi, Cristina Baldoli, Sabrina Canale, Maria Sessa, Patrizia D’Adamo, Alessandro Aiuti.
Scientific Reports. 2017; 7:40136.
Cicalese MP, Ferrua F, Castagnaro L, Pajno R, Barzaghi F, Giannelli S, Dionisio F, Brigida I, Bonopane M, Casiraghi M, Tabucchi A, Carlucci F, Grunebaum E, Adeli M, Bredius RG, Puck JM, Stepensky P, Tezcan I, Rolfe K, De Boever E, Reinhardt RR, Appleby J, Ciceri F, Roncarolo MG, Aiuti A.
Blood. 2016; 128:45-54.
A 24 year enzyme replacement therapy in an adenosine-deaminase-deficient patient.
Tartibi HM, Hershfield MS, Bahna SL.
Pediatrics. 2016; 137:e20152169 (DOI: 10.1542/peds.2015-2169 )
Komarow HD, Sokolic R, Hershfield MS, Kohn DB, Young M, Metcalfe DD, Candotti F.
Orphanet J Rare Dis. 2015; 10:159.
Baffelli R, Notarangelo LD, Imberti L, Hershfield MS, Serana F, Santisteban I, Bolda F, Porta F, Lanfranchi A.
J Clin Immunol. 2015; 35:624-37.
Adams SP, Wilson M, Harb E, Fairbanks L, Xu-Bayford J, Brown L, Kearney L, Madkaikar M, Bobby Gaspar H.
Clin Immunol. 2015; 161:174-9.
Nikolajeva O, Worth A, Hague R, Martinez-Alier N, Smart J, Adams S, Davies EG, Gaspar HB.
J Clin Immunol. 2015; 35:366-72.
Otsu M, Yamada M, Nakajima S, Kida M, Maeyama Y, Hatano N, Toita N, Takezaki S, Okura Y, Kobayashi R, Matsumoto Y, Tatsuzawa O, Tsuchida F, Kato S, Kitagawa M, Mineno J, Hershfield MS, Bali P, Candotti F, Onodera M, Kawamura N, Sakiyama Y, Ariga T.
J Clin Immunol. 2015; 35:384-98.
The inclusion of ADA-SCID in expanded newborn screening by tandem mass spectrometry.
la Marca G, Giocaliere E, Malvagia S, Funghini S, Ombrone D, Della Bona ML, Canessa C, Lippi F, Romano F, Guerrini R, Resti M, Azzari C.
J Pharm Biomed Anal. 2014; 88:201-6.
Carbonaro DA, Zhang L, Jin X, Montiel-Equihua C, Geiger S, Carmo M, Cooper A, Fairbanks L, Kaufman ML, Sebire NJ, Hollis RP, Blundell MP, Senadheera S, Fu PY, Sahaghian A, Chan RY, Wang X, Cornetta K, Thrasher AJ, Kohn DB, Gaspar HB.
Mol Ther. 2014; 22:607-22.
la Marca G, Canessa C, Giocaliere E, Romano F, Duse M, Malvagia S, Lippi F, Funghini S, Bianchi L, Della Bona ML, Valleriani C, Ombrone D, Moriondo M, Villanelli F, Speckmann C, Adams S, Gaspar BH, Hershfield M, Santisteban I, Fairbanks L, Ragusa G, Resti M, de Martino M, Guerrini R, Azzari C.
J Allergy Clin Immunol. 2013; 131:1604-10.
Geelen J, Pfundt R, Meijer J, Verheijen FW, van Kuilenburg AB, Warris A, Marcelis C.
J Allergy Clin Immunol. 2013; 132:222-3.
Pulmonary alveolar proteinosis in patients with adenosine deaminase deficiency.
Grunebaum E, Cutz E, Roifman CM.
J Allergy Clin Immunol. 2012;129(6):1588-1593
C. Booth, V. Algar, J. Xu-Bayford, L. Fairbanks, C. Owens, H. Gaspar
J Clin Immunol. 2012; 32:449-453
Hassan A, Booth C, Brightwell A, Allwood Z, Veys P, Rao K, Hönig M, Friedrich W, Gennery A, Slatter M, Bredius R, Finocchi A, Cancrini C, Aiuti A, Porta F, Lanfranchi A, Ridella M, Steward C, Filipovich A, Marsh R, Bordon V, Al-Muhsen S, Al-Mousa H, Alsum Z, Al-Dhekri H, Al Ghonaium A, Speckmann C, Fischer A, Mahlaoui N, Nichols KE, Grunebaum E, Al Zahrani D, Roifman CM, Boelens J, Davies EG, Cavazzana-Calvo M, Notarangelo L, Gaspar HB; Inborn Errors Working Party of the European Group for Blood and Marrow Transplantation and European Society for Immunodeficiency.
Blood 2012; 120:3615-3624
Rogers MH1, Lwin R, Fairbanks L, Gerritsen B, Gaspar HB.
J Pediatr. 2001; 139(1):44-50.