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446 results

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Page 1
Gene therapy in rare diseases: the benefits and challenges of developing a patient-centric registry for Strimvelis in ADA-SCID.
Stirnadel-Farrant H, Kudari M, Garman N, Imrie J, Chopra B, Giannelli S, Gabaldo M, Corti A, Zancan S, Aiuti A, Cicalese MP, Batta R, Appleby J, Davinelli M, Ng P. Stirnadel-Farrant H, et al. Among authors: appleby j. Orphanet J Rare Dis. 2018 Apr 6;13(1):49. doi: 10.1186/s13023-018-0791-9. Orphanet J Rare Dis. 2018. PMID: 29625577 Free PMC article.
Update on the safety and efficacy of retroviral gene therapy for immunodeficiency due to adenosine deaminase deficiency.
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. Cicalese MP, et al. Among authors: appleby j. Blood. 2016 Jul 7;128(1):45-54. doi: 10.1182/blood-2016-01-688226. Epub 2016 Apr 29. Blood. 2016. PMID: 27129325 Free PMC article. Clinical Trial.
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: appleby j. 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.
Good Laboratory Practice Preclinical Safety Studies for GSK2696273 (MLV Vector-Based Ex Vivo Gene Therapy for Adenosine Deaminase Deficiency Severe Combined Immunodeficiency) in NSG Mice.
Carriglio N, Klapwijk J, Hernandez RJ, Vezzoli M, Chanut F, Lowe R, Draghici E, Nord M, Albertini P, Cristofori P, Richards J, Staton H, Appleby J, Aiuti A, Sauer AV. Carriglio N, et al. Among authors: appleby j. Hum Gene Ther Clin Dev. 2017 Mar;28(1):17-27. doi: 10.1089/humc.2016.191. Hum Gene Ther Clin Dev. 2017. PMID: 28319446
How Europe copes with flu.
Appleby J. Appleby J. BMJ. 2018 Jan 25;360:k266. doi: 10.1136/bmj.k266. BMJ. 2018. PMID: 29371180 No abstract available.
What is happening to non-covid deaths?
Appleby J. Appleby J. BMJ. 2020 Apr 24;369:m1607. doi: 10.1136/bmj.m1607. BMJ. 2020. PMID: 32332059 No abstract available.
446 results