214 related articles for article (PubMed ID: 20354504)
1. 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]
2. 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]
3. Correction of SCID-X1 using an enhancerless Vav promoter.
Almarza E; Zhang F; Santilli G; Blundell MP; Howe SJ; Thornhill SI; Bueren JA; Thrasher AJ
Hum Gene Ther; 2011 Mar; 22(3):263-70. PubMed ID: 20887212
[TBL] [Abstract][Full Text] [Related]
4. 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]
5. 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]
6. 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]
7. 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]
8. Correction of murine SCID-X1 by lentiviral gene therapy using a codon-optimized IL2RG gene and minimal pretransplant conditioning.
Huston MW; van Til NP; Visser TP; Arshad S; Brugman MH; Cattoglio C; Nowrouzi A; Li Y; Schambach A; Schmidt M; Baum C; von Kalle C; Mavilio F; Zhang F; Blundell MP; Thrasher AJ; Verstegen MM; Wagemaker G
Mol Ther; 2011 Oct; 19(10):1867-77. PubMed ID: 21750532
[TBL] [Abstract][Full Text] [Related]
9. 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]
10. 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]
11. 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]
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. Lentiviral vectors containing an enhancer-less ubiquitously acting chromatin opening element (UCOE) provide highly reproducible and stable transgene expression in hematopoietic cells.
Zhang F; Thornhill SI; Howe SJ; Ulaganathan M; Schambach A; Sinclair J; Kinnon C; Gaspar HB; Antoniou M; Thrasher AJ
Blood; 2007 Sep; 110(5):1448-57. PubMed ID: 17456723
[TBL] [Abstract][Full Text] [Related]
14.
Rajawat YS; Humbert O; Cook SM; Radtke S; Pande D; Enstrom M; Wohlfahrt ME; Kiem HP
Hum Gene Ther; 2021 Jan; 32(1-2):113-127. PubMed ID: 32741228
[TBL] [Abstract][Full Text] [Related]
15. Ten years of gene therapy for primary immune deficiencies.
Aiuti A; Roncarolo MG
Hematology Am Soc Hematol Educ Program; 2009; ():682-9. PubMed ID: 20008254
[TBL] [Abstract][Full Text] [Related]
16. 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]
17. 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]
18. Insertional gene activation by lentiviral and gammaretroviral vectors.
Bokhoven M; Stephen SL; Knight S; Gevers EF; Robinson IC; Takeuchi Y; Collins MK
J Virol; 2009 Jan; 83(1):283-94. PubMed ID: 18945765
[TBL] [Abstract][Full Text] [Related]
19. Limiting Thymic Precursor Supply Increases the Risk of Lymphoid Malignancy in Murine X-Linked Severe Combined Immunodeficiency.
Ginn SL; Hallwirth CV; Liao SHY; Teber ET; Arthur JW; Wu J; Lee HC; Tay SS; Hu M; Reddel RR; McCormack MP; Thrasher AJ; Cavazzana M; Alexander SI; Alexander IE
Mol Ther Nucleic Acids; 2017 Mar; 6():1-14. PubMed ID: 28325276
[TBL] [Abstract][Full Text] [Related]
20. Lentiviral vector transduction of NOD/SCID repopulating cells results in multiple vector integrations per transduced cell: risk of insertional mutagenesis.
Woods NB; Muessig A; Schmidt M; Flygare J; Olsson K; Salmon P; Trono D; von Kalle C; Karlsson S
Blood; 2003 Feb; 101(4):1284-9. PubMed ID: 12393514
[TBL] [Abstract][Full Text] [Related]
[Next] [New Search]
