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Results: 1 to 20 of 102

References for PMC Articles for PubMed (Select 21261461)

1.

Stem-cell gene therapy for the Wiskott-Aldrich syndrome.

Boztug K, Schmidt M, Schwarzer A, Banerjee PP, Díez IA, Dewey RA, Böhm M, Nowrouzi A, Ball CR, Glimm H, Naundorf S, Kühlcke K, Blasczyk R, Kondratenko I, Maródi L, Orange JS, von Kalle C, Klein C.

N Engl J Med. 2010 Nov 11;363(20):1918-27. doi: 10.1056/NEJMoa1003548.

2.

Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia.

Cavazzana-Calvo M, Payen E, Negre O, Wang G, Hehir K, Fusil F, Down J, Denaro M, Brady T, Westerman K, Cavallesco R, Gillet-Legrand B, Caccavelli L, Sgarra R, Maouche-Chrétien L, Bernaudin F, Girot R, Dorazio R, Mulder GJ, Polack A, Bank A, Soulier J, Larghero J, Kabbara N, Dalle B, Gourmel B, Socie G, Chrétien S, Cartier N, Aubourg P, Fischer A, Cornetta K, Galacteros F, Beuzard Y, Gluckman E, Bushman F, Hacein-Bey-Abina S, Leboulch P.

Nature. 2010 Sep 16;467(7313):318-22. doi: 10.1038/nature09328.

3.

Efficient and stable MGMT-mediated selection of long-term repopulating stem cells in nonhuman primates.

Beard BC, Trobridge GD, Ironside C, McCune JS, Adair JE, Kiem HP.

J Clin Invest. 2010 Jul;120(7):2345-54. doi: 10.1172/JCI40767. Epub 2010 Jun 14.

4.

A transposon and transposase system for human application.

Hackett PB, Largaespada DA, Cooper LJ.

Mol Ther. 2010 Apr;18(4):674-83. doi: 10.1038/mt.2010.2. Epub 2010 Jan 26. Review.

5.

Cellular barcoding tool for clonal analysis in the hematopoietic system.

Gerrits A, Dykstra B, Kalmykowa OJ, Klauke K, Verovskaya E, Broekhuis MJ, de Haan G, Bystrykh LV.

Blood. 2010 Apr 1;115(13):2610-8. doi: 10.1182/blood-2009-06-229757. Epub 2010 Jan 21.

6.

Lentiviral gene ontology (LeGO) vectors equipped with novel drug-selectable fluorescent proteins: new building blocks for cell marking and multi-gene analysis.

Weber K, Mock U, Petrowitz B, Bartsch U, Fehse B.

Gene Ther. 2010 Apr;17(4):511-20. doi: 10.1038/gt.2009.149. Epub 2009 Dec 17.

PMID:
20016542
7.

Comprehensive genomic access to vector integration in clinical gene therapy.

Gabriel R, Eckenberg R, Paruzynski A, Bartholomae CC, Nowrouzi A, Arens A, Howe SJ, Recchia A, Cattoglio C, Wang W, Faber K, Schwarzwaelder K, Kirsten R, Deichmann A, Ball CR, Balaggan KS, Yáñez-Muñoz RJ, Ali RR, Gaspar HB, Biasco L, Aiuti A, Cesana D, Montini E, Naldini L, Cohen-Haguenauer O, Mavilio F, Thrasher AJ, Glimm H, von Kalle C, Saurin W, Schmidt M.

Nat Med. 2009 Dec;15(12):1431-6. doi: 10.1038/nm.2057. Epub 2009 Nov 22.

PMID:
19966782
8.

Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy.

Cartier N, Hacein-Bey-Abina S, Bartholomae CC, Veres G, Schmidt M, Kutschera I, Vidaud M, Abel U, Dal-Cortivo L, Caccavelli L, Mahlaoui N, Kiermer V, Mittelstaedt D, Bellesme C, Lahlou N, Lefrère F, Blanche S, Audit M, Payen E, Leboulch P, l'Homme B, Bougnères P, Von Kalle C, Fischer A, Cavazzana-Calvo M, Aubourg P.

Science. 2009 Nov 6;326(5954):818-23. doi: 10.1126/science.1171242.

9.

Long-term outcome of EBV-specific T-cell infusions to prevent or treat EBV-related lymphoproliferative disease in transplant recipients.

Heslop HE, Slobod KS, Pule MA, Hale GA, Rousseau A, Smith CA, Bollard CM, Liu H, Wu MF, Rochester RJ, Amrolia PJ, Hurwitz JL, Brenner MK, Rooney CM.

Blood. 2010 Feb 4;115(5):925-35. doi: 10.1182/blood-2009-08-239186. Epub 2009 Oct 30.

10.

Stable transgene expression in primitive human CD34+ hematopoietic stem/progenitor cells, using the Sleeping Beauty transposon system.

Sumiyoshi T, Holt NG, Hollis RP, Ge S, Cannon PM, Crooks GM, Kohn DB.

Hum Gene Ther. 2009 Dec;20(12):1607-26. doi: 10.1089/hum.2009.109.

11.

Integration of retroviral vectors induces minor changes in the transcriptional activity of T cells from ADA-SCID patients treated with gene therapy.

Cassani B, Montini E, Maruggi G, Ambrosi A, Mirolo M, Selleri S, Biral E, Frugnoli I, Hernandez-Trujillo V, Di Serio C, Roncarolo MG, Naldini L, Mavilio F, Aiuti A.

Blood. 2009 Oct 22;114(17):3546-56. doi: 10.1182/blood-2009-02-202085. Epub 2009 Aug 3.

12.

Self-inactivating retroviral vector-mediated gene transfer induces oncogene activation and immortalization of primary murine bone marrow cells.

Bosticardo M, Ghosh A, Du Y, Jenkins NA, Copeland NG, Candotti F.

Mol Ther. 2009 Nov;17(11):1910-8. doi: 10.1038/mt.2009.172. Epub 2009 Jul 28.

13.

In vivo selection of hematopoietic progenitor cells and temozolomide dose intensification in rhesus macaques through lentiviral transduction with a drug resistance gene.

Larochelle A, Choi U, Shou Y, Naumann N, Loktionova NA, Clevenger JR, Krouse A, Metzger M, Donahue RE, Kang E, Stewart C, Persons D, Malech HL, Dunbar CE, Sorrentino BP.

J Clin Invest. 2009 Jul;119(7):1952-63. doi: 10.1172/JCI37506. Epub 2009 Jun 8.

14.

Stable nonviral gene transfer into primary human T cells.

Magg T, Hartrampf S, Albert MH.

Hum Gene Ther. 2009 Sep;20(9):989-98. doi: 10.1089/hum.2008.180.

PMID:
19485761
15.

Stable gene transfer and expression in cord blood-derived CD34+ hematopoietic stem and progenitor cells by a hyperactive Sleeping Beauty transposon system.

Xue X, Huang X, Nodland SE, Mátés L, Ma L, Izsvák Z, Ivics Z, LeBien TW, McIvor RS, Wagner JE, Zhou X.

Blood. 2009 Aug 13;114(7):1319-30. doi: 10.1182/blood-2009-03-210005. Epub 2009 May 4.

16.

Sustained high-level polyclonal hematopoietic marking and transgene expression 4 years after autologous transplantation of rhesus macaques with SIV lentiviral vector-transduced CD34+ cells.

Kim YJ, Kim YS, Larochelle A, Renaud G, Wolfsberg TG, Adler R, Donahue RE, Hematti P, Hong BK, Roayaei J, Akagi K, Riberdy JM, Nienhuis AW, Dunbar CE, Persons DA.

Blood. 2009 May 28;113(22):5434-43. doi: 10.1182/blood-2008-10-185199. Epub 2009 Apr 1.

17.

Adoptive cell therapy for the treatment of patients with metastatic melanoma.

Rosenberg SA, Dudley ME.

Curr Opin Immunol. 2009 Apr;21(2):233-40. doi: 10.1016/j.coi.2009.03.002. Epub 2009 Mar 21. Review.

18.

Transient in vivo beta-globin production after lentiviral gene transfer to hematopoietic stem cells in the nonhuman primate.

Hayakawa J, Ueda T, Lisowski L, Hsieh MM, Washington K, Phang O, Metzger M, Krouse A, Donahue RE, Sadelain M, Tisdale JF.

Hum Gene Ther. 2009 Jun;20(6):563-72. doi: 10.1089/hum.2008.186.

19.

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 29;360(5):447-58. doi: 10.1056/NEJMoa0805817.

20.

Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients.

Howe SJ, Mansour MR, Schwarzwaelder K, Bartholomae C, Hubank M, Kempski H, Brugman MH, Pike-Overzet K, Chatters SJ, de Ridder D, Gilmour KC, Adams S, Thornhill SI, Parsley KL, Staal FJ, Gale RE, Linch DC, Bayford J, Brown L, Quaye M, Kinnon C, Ancliff P, Webb DK, Schmidt M, von Kalle C, Gaspar HB, Thrasher AJ.

J Clin Invest. 2008 Sep;118(9):3143-50. doi: 10.1172/JCI35798.

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