These tools will no longer be maintained as of December 31, 2024. Archived website can be found here. PubMed4Hh GitHub repository can be found here. Contact NLM Customer Service if you have questions.
2. Gene Therapy for X-Linked Severe Combined Immunodeficiency: Where Do We Stand? Cavazzana M; Six E; Lagresle-Peyrou C; André-Schmutz I; Hacein-Bey-Abina S Hum Gene Ther; 2016 Feb; 27(2):108-16. PubMed ID: 26790362 [TBL] [Abstract][Full Text] [Related]
4. A modified γ-retrovirus vector for X-linked severe combined immunodeficiency. Hacein-Bey-Abina S; Pai SY; Gaspar HB; Armant M; Berry CC; Blanche S; Bleesing J; Blondeau J; de Boer H; Buckland KF; Caccavelli L; Cros G; De Oliveira S; Fernández KS; Guo D; Harris CE; Hopkins G; Lehmann LE; Lim A; London WB; van der Loo JC; Malani N; Male F; Malik P; Marinovic MA; McNicol AM; Moshous D; Neven B; Oleastro M; Picard C; Ritz J; Rivat C; Schambach A; Shaw KL; Sherman EA; Silberstein LE; Six E; Touzot F; Tsytsykova A; Xu-Bayford J; Baum C; Bushman FD; Fischer A; Kohn DB; Filipovich AH; Notarangelo LD; Cavazzana M; Williams DA; Thrasher AJ N Engl J Med; 2014 Oct; 371(15):1407-17. PubMed ID: 25295500 [TBL] [Abstract][Full Text] [Related]
5. Lentiviral hematopoietic stem cell gene therapy for X-linked severe combined immunodeficiency. De Ravin SS; Wu X; Moir S; Anaya-O'Brien S; Kwatemaa N; Littel P; Theobald N; Choi U; Su L; Marquesen M; Hilligoss D; Lee J; Buckner CM; Zarember KA; O'Connor G; McVicar D; Kuhns D; Throm RE; Zhou S; Notarangelo LD; Hanson IC; Cowan MJ; Kang E; Hadigan C; Meagher M; Gray JT; Sorrentino BP; Malech HL; Kardava L Sci Transl Med; 2016 Apr; 8(335):335ra57. PubMed ID: 27099176 [TBL] [Abstract][Full Text] [Related]
6. Lentiviral Gene Therapy Combined with Low-Dose Busulfan in Infants with SCID-X1. Mamcarz E; Zhou S; Lockey T; Abdelsamed H; Cross SJ; Kang G; Ma Z; Condori J; Dowdy J; Triplett B; Li C; Maron G; Aldave Becerra JC; Church JA; Dokmeci E; Love JT; da Matta Ain AC; van der Watt H; Tang X; Janssen W; Ryu BY; De Ravin SS; Weiss MJ; Youngblood B; Long-Boyle JR; Gottschalk S; Meagher MM; Malech HL; Puck JM; Cowan MJ; Sorrentino BP N Engl J Med; 2019 Apr; 380(16):1525-1534. PubMed ID: 30995372 [TBL] [Abstract][Full Text] [Related]
7. Vector integration is nonrandom and clustered and influences the fate of lymphopoiesis in SCID-X1 gene therapy. Deichmann A; Hacein-Bey-Abina S; Schmidt M; Garrigue A; Brugman MH; Hu J; Glimm H; Gyapay G; Prum B; Fraser CC; Fischer N; Schwarzwaelder K; Siegler ML; de Ridder D; Pike-Overzet K; Howe SJ; Thrasher AJ; Wagemaker G; Abel U; Staal FJ; Delabesse E; Villeval JL; Aronow B; Hue C; Prinz C; Wissler M; Klanke C; Weissenbach J; Alexander I; Fischer A; von Kalle C; Cavazzana-Calvo M J Clin Invest; 2007 Aug; 117(8):2225-32. PubMed ID: 17671652 [TBL] [Abstract][Full Text] [Related]
8. Critical variables affecting clinical-grade production of the self-inactivating gamma-retroviral vector for the treatment of X-linked severe combined immunodeficiency. van der Loo JC; Swaney WP; Grassman E; Terwilliger A; Higashimoto T; Schambach A; Hacein-Bey-Abina S; Nordling DL; Cavazzana-Calvo M; Thrasher AJ; Williams DA; Reeves L; Malik P Gene Ther; 2012 Aug; 19(8):872-6. PubMed ID: 22551777 [TBL] [Abstract][Full Text] [Related]
9. A novel model of SCID-X1 reconstitution reveals predisposition to retrovirus-induced lymphoma but no evidence of gammaC gene oncogenicity. Scobie L; Hector RD; Grant L; Bell M; Nielsen AA; Meikle S; Philbey A; Thrasher AJ; Cameron ER; Blyth K; Neil JC Mol Ther; 2009 Jun; 17(6):1031-8. PubMed ID: 19337236 [TBL] [Abstract][Full Text] [Related]
10. Gene therapy model of X-linked severe combined immunodeficiency using a modified foamy virus vector. Horino S; Uchiyama T; So T; Nagashima H; Sun SL; Sato M; Asao A; Haji Y; Sasahara Y; Candotti F; Tsuchiya S; Kure S; Sugamura K; Ishii N PLoS One; 2013; 8(8):e71594. PubMed ID: 23990961 [TBL] [Abstract][Full Text] [Related]
11. Dynamics of gene-modified progenitor cells analyzed by tracking retroviral integration sites in a human SCID-X1 gene therapy trial. Wang GP; Berry CC; Malani N; Leboulch P; Fischer A; Hacein-Bey-Abina S; Cavazzana-Calvo M; Bushman FD Blood; 2010 Jun; 115(22):4356-66. PubMed ID: 20228274 [TBL] [Abstract][Full Text] [Related]
12. 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]
13. Retrovirus-induced oncogenesis and safety of retroviral vectors. Nair V Curr Opin Mol Ther; 2008 Oct; 10(5):431-8. PubMed ID: 18830918 [TBL] [Abstract][Full Text] [Related]
14. Lymphomagenesis in SCID-X1 mice following lentivirus-mediated phenotype correction independent of insertional mutagenesis and gammac overexpression. Ginn SL; Liao SH; Dane AP; Hu M; Hyman J; Finnie JW; Zheng M; Cavazzana-Calvo M; Alexander SI; Thrasher AJ; Alexander IE Mol Ther; 2010 May; 18(5):965-76. PubMed ID: 20354504 [TBL] [Abstract][Full Text] [Related]
15. Long-term persistence of a polyclonal T cell repertoire after gene therapy for X-linked severe combined immunodeficiency. Gaspar HB; Cooray S; Gilmour KC; Parsley KL; Adams S; Howe SJ; Al Ghonaium A; Bayford J; Brown L; Davies EG; Kinnon C; Thrasher AJ Sci Transl Med; 2011 Aug; 3(97):97ra79. PubMed ID: 21865537 [TBL] [Abstract][Full Text] [Related]
16. Pretransplant mobilization with granulocyte colony-stimulating factor improves B-cell reconstitution by lentiviral vector gene therapy in SCID-X1 mice. Huston MW; Riegman AR; Yadak R; van Helsdingen Y; de Boer H; van Til NP; Wagemaker G Hum Gene Ther; 2014 Oct; 25(10):905-14. PubMed ID: 25222508 [TBL] [Abstract][Full Text] [Related]
17. Self-inactivating gammaretroviral vectors for gene therapy of X-linked severe combined immunodeficiency. Thornhill SI; Schambach A; Howe SJ; Ulaganathan M; Grassman E; Williams D; Schiedlmeier B; Sebire NJ; Gaspar HB; Kinnon C; Baum C; Thrasher AJ Mol Ther; 2008 Mar; 16(3):590-8. PubMed ID: 18180772 [TBL] [Abstract][Full Text] [Related]
18. Transduction of human CD34+ repopulating cells with a self-inactivating lentiviral vector for SCID-X1 produced at clinical scale by a stable cell line. Greene MR; Lockey T; Mehta PK; Kim YS; Eldridge PW; Gray JT; Sorrentino BP Hum Gene Ther Methods; 2012 Oct; 23(5):297-308. PubMed ID: 23075105 [TBL] [Abstract][Full Text] [Related]
19. Murine leukemias with retroviral insertions at Lmo2 are predictive of the leukemias induced in SCID-X1 patients following retroviral gene therapy. Davé UP; Akagi K; Tripathi R; Cleveland SM; Thompson MA; Yi M; Stephens R; Downing JR; Jenkins NA; Copeland NG PLoS Genet; 2009 May; 5(5):e1000491. PubMed ID: 19461887 [TBL] [Abstract][Full Text] [Related]
20. Review: Oncogenic Insertional Mutagenesis as a Consequence of Retroviral Gene Therapy for X-Linked Severe Combined Immunodeficiency Disease. Ahmed B; Zafar M; Qadir MI Crit Rev Eukaryot Gene Expr; 2019; 29(6):511-520. PubMed ID: 32422006 [TBL] [Abstract][Full Text] [Related] [Next] [New Search]