325 related articles for article (PubMed ID: 28351939)
21. Gene therapy's out-of-body experience.
Scott CT; DeFrancesco L
Nat Biotechnol; 2016 Jun; 34(6):600-7. PubMed ID: 27281416
[No Abstract] [Full Text] [Related]
22. Promising Results with Ex Vivo Lentiviral HSPC Gene Therapy in ADA-SCID.
Am J Med Genet A; 2021 Aug; 185(8):2291-2292. PubMed ID: 34240545
[No Abstract] [Full Text] [Related]
23. Successful reconstitution of immunity in ADA-SCID by stem cell gene therapy following cessation of PEG-ADA and use of mild preconditioning.
Gaspar HB; Bjorkegren E; Parsley K; Gilmour KC; King D; Sinclair J; Zhang F; Giannakopoulos A; Adams S; Fairbanks LD; Gaspar J; Henderson L; Xu-Bayford JH; Davies EG; Veys PA; Kinnon C; Thrasher AJ
Mol Ther; 2006 Oct; 14(4):505-13. PubMed ID: 16905365
[TBL] [Abstract][Full Text] [Related]
24. Investor Outlook: Rising from the Ashes; GSK's European Approval of Strimvelis for ADA-SCID.
Schimmer J; Breazzano S
Hum Gene Ther Clin Dev; 2016 Jun; 27(2):57-61. PubMed ID: 27267267
[TBL] [Abstract][Full Text] [Related]
25. Towards a rAAV-based gene therapy for ADA-SCID: from ADA deficiency to current and future treatment strategies.
Silver JN; Flotte TR
Pharmacogenomics; 2008 Jul; 9(7):947-68. PubMed ID: 18597656
[TBL] [Abstract][Full Text] [Related]
26. Gene therapy for adenosine deaminase-deficient severe combined immune deficiency: clinical comparison of retroviral vectors and treatment plans.
Candotti F; Shaw KL; Muul L; Carbonaro D; Sokolic R; Choi C; Schurman SH; Garabedian E; Kesserwan C; Jagadeesh GJ; Fu PY; Gschweng E; Cooper A; Tisdale JF; Weinberg KI; Crooks GM; Kapoor N; Shah A; Abdel-Azim H; Yu XJ; Smogorzewska M; Wayne AS; Rosenblatt HM; Davis CM; Hanson C; Rishi RG; Wang X; Gjertson D; Yang OO; Balamurugan A; Bauer G; Ireland JA; Engel BC; Podsakoff GM; Hershfield MS; Blaese RM; Parkman R; Kohn DB
Blood; 2012 Nov; 120(18):3635-46. PubMed ID: 22968453
[TBL] [Abstract][Full Text] [Related]
27. Gene therapy for PIDs: progress, pitfalls and prospects.
Mukherjee S; Thrasher AJ
Gene; 2013 Aug; 525(2):174-81. PubMed ID: 23566838
[TBL] [Abstract][Full Text] [Related]
28. Hematopoietic stem cell gene therapy for adenosine deaminase deficient-SCID.
Aiuti A; Brigida I; Ferrua F; Cappelli B; Chiesa R; Marktel S; Roncarolo MG
Immunol Res; 2009; 44(1-3):150-9. PubMed ID: 19224139
[TBL] [Abstract][Full Text] [Related]
29. 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
N Engl J Med; 2009 Jan; 360(5):447-58. PubMed ID: 19179314
[TBL] [Abstract][Full Text] [Related]
30. Gene-transferred oligoclonal T cells predominantly persist in peripheral blood from an adenosine deaminase-deficient patient during gene therapy.
Misaki Y; Ezaki I; Ariga T; Kawamura N; Sakiyama Y; Yamamoto K
Mol Ther; 2001 Jan; 3(1):24-7. PubMed ID: 11162307
[TBL] [Abstract][Full Text] [Related]
31. Treatment of severe combined immunodeficiency disease (SCID) due to adenosine deaminase deficiency with CD34+ selected autologous peripheral blood cells transduced with a human ADA gene. Amendment to clinical research project, Project 90-C-195, January 10, 1992.
Blaese RM; Culver KW; Chang L; Anderson WF; Mullen C; Nienhuis A; Carter C; Dunbar C; Leitman S; Berger M
Hum Gene Ther; 1993 Aug; 4(4):521-7. PubMed ID: 7691188
[TBL] [Abstract][Full Text] [Related]
32. A self-inactivating lentiviral vector for SCID-X1 gene therapy that does not activate LMO2 expression in human T cells.
Zhou S; Mody D; DeRavin SS; Hauer J; Lu T; Ma Z; Hacein-Bey Abina S; Gray JT; Greene MR; Cavazzana-Calvo M; Malech HL; Sorrentino BP
Blood; 2010 Aug; 116(6):900-8. PubMed ID: 20457870
[TBL] [Abstract][Full Text] [Related]
33. Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1.
Hacein-Bey-Abina S; Garrigue A; Wang GP; Soulier J; Lim A; Morillon E; Clappier E; Caccavelli L; Delabesse E; Beldjord K; Asnafi V; MacIntyre E; Dal Cortivo L; Radford I; Brousse N; Sigaux F; Moshous D; Hauer J; Borkhardt A; Belohradsky BH; Wintergerst U; Velez MC; Leiva L; Sorensen R; Wulffraat N; Blanche S; Bushman FD; Fischer A; Cavazzana-Calvo M
J Clin Invest; 2008 Sep; 118(9):3132-42. PubMed ID: 18688285
[TBL] [Abstract][Full Text] [Related]
34. 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
Nat Med; 2024 Feb; 30(2):488-497. PubMed ID: 38355973
[TBL] [Abstract][Full Text] [Related]
35. Evaluation of ADA gene expression and transduction efficiency in ADA/SCID patients undergoing gene therapy.
Carlucci F; Tabucchi A; Aiuti A; Rosi F; Floccari F; Pagani R; Marinello E
Nucleosides Nucleotides Nucleic Acids; 2004 Oct; 23(8-9):1245-8. PubMed ID: 15571238
[TBL] [Abstract][Full Text] [Related]
36. Gene therapy for severe combined immunodeficiencies and beyond.
Fischer A; Hacein-Bey-Abina S
J Exp Med; 2020 Jan; 217(2):. PubMed ID: 31826240
[TBL] [Abstract][Full Text] [Related]
37. [Transplantation of genetically modified cells in the treatment of children with SCID: great hopes and recent disappointments].
Smogorzewska EM; Weinberg KI; Kohn DB
Med Wieku Rozwoj; 2003; 7(1):27-34. PubMed ID: 13130167
[TBL] [Abstract][Full Text] [Related]
38. Gene therapy/bone marrow transplantation in ADA-deficient mice: roles of enzyme-replacement therapy and cytoreduction.
Carbonaro DA; Jin X; Wang X; Yu XJ; Rozengurt N; Kaufman ML; Wang X; Gjertson D; Zhou Y; Blackburn MR; Kohn DB
Blood; 2012 Nov; 120(18):3677-87. PubMed ID: 22833548
[TBL] [Abstract][Full Text] [Related]
39. Busulfan Pharmacokinetics in Adenosine Deaminase-Deficient Severe Combined Immunodeficiency Gene Therapy.
Bradford KL; Liu S; Krajinovic M; Ansari M; Garabedian E; Tse J; Wang X; Shaw KL; Gaspar HB; Candotti F; Kohn DB
Biol Blood Marrow Transplant; 2020 Oct; 26(10):1819-1827. PubMed ID: 32653625
[TBL] [Abstract][Full Text] [Related]
40. Gene therapy for severe combined immunodeficiency caused by adenosine deaminase deficiency: improved retroviral vectors for clinical trials.
Onodera M; Nelson DM; Sakiyama Y; Candotti F; Blaese RM
Acta Haematol; 1999; 101(2):89-96. PubMed ID: 10202239
[TBL] [Abstract][Full Text] [Related]
[Previous] [Next] [New Search]
