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Items: 1 to 20 of 94

1.

Gene therapy for Fanconi anemia: one step closer to the clinic.

Tolar J, Becker PS, Clapp DW, Hanenberg H, de Heredia CD, Kiem HP, Navarro S, Qasba P, Rio P, Schmidt M, Sevilla J, Verhoeyen E, Thrasher AJ, Bueren J.

Hum Gene Ther. 2012 Feb;23(2):141-4. doi: 10.1089/hum.2011.237. No abstract available.

2.

Lentiviral-Mediated Gene Therapy in Fanconi Anemia-A Mice Reveals Long-Term Engraftment and Continuous Turnover of Corrected HSCs.

Molina-Estevez FJ, Nowrouzi A, Lozano ML, Galy A, Charrier S, von Kalle C, Guenechea G, Bueren JA, Schmidt M.

Curr Gene Ther. 2015;15(6):550-62.

PMID:
26415575
3.

Preclinical correction of human Fanconi anemia complementation group A bone marrow cells using a safety-modified lentiviral vector.

Becker PS, Taylor JA, Trobridge GD, Zhao X, Beard BC, Chien S, Adair J, Kohn DB, Wagner JE, Shimamura A, Kiem HP.

Gene Ther. 2010 Oct;17(10):1244-52. doi: 10.1038/gt.2010.62. Epub 2010 May 20.

4.

Targeted gene therapy and cell reprogramming in Fanconi anemia.

Rio P, Baños R, Lombardo A, Quintana-Bustamante O, Alvarez L, Garate Z, Genovese P, Almarza E, Valeri A, Díez B, Navarro S, Torres Y, Trujillo JP, Murillas R, Segovia JC, Samper E, Surralles J, Gregory PD, Holmes MC, Naldini L, Bueren JA.

EMBO Mol Med. 2014 Jun;6(6):835-48. doi: 10.15252/emmm.201303374. Epub 2014 Apr 6.

5.
6.

Overcoming reprogramming resistance of Fanconi anemia cells.

Müller LU, Milsom MD, Harris CE, Vyas R, Brumme KM, Parmar K, Moreau LA, Schambach A, Park IH, London WB, Strait K, Schlaeger T, Devine AL, Grassman E, D'Andrea A, Daley GQ, Williams DA.

Blood. 2012 Jun 7;119(23):5449-57. doi: 10.1182/blood-2012-02-408674. Epub 2012 Feb 27.

7.

[Fanconi's pancytopenia syndrome].

Higuchi T, Koike K.

Nihon Rinsho. 2006 Sep 28;Suppl 3:418-21. Review. Japanese. No abstract available.

PMID:
17022578
8.

[Recent progress in understanding molecular pathogenesis of Fanconi anemia].

Yamashita T, Oda T, Sekimoto T.

Rinsho Ketsueki. 2009 Jul;50(7):538-46. Review. Japanese. No abstract available.

PMID:
19638721
9.

Continuous in vivo infusion of interferon-gamma (IFN-gamma) enhances engraftment of syngeneic wild-type cells in Fanca-/- and Fancg-/- mice.

Si Y, Ciccone S, Yang FC, Yuan J, Zeng D, Chen S, van de Vrugt HJ, Critser J, Arwert F, Haneline LS, Clapp DW.

Blood. 2006 Dec 15;108(13):4283-7. Epub 2006 Aug 31.

10.

Current knowledge on the pathophysiology of Fanconi anemia: from genes to phenotypes.

Yamashita T, Nakahata T.

Int J Hematol. 2001 Jul;74(1):33-41. Review.

PMID:
11530803
11.

Rapid lentiviral transduction preserves the engraftment potential of Fanca(-/-) hematopoietic stem cells.

Müller LU, Milsom MD, Kim MO, Schambach A, Schuesler T, Williams DA.

Mol Ther. 2008 Jun;16(6):1154-60. doi: 10.1038/mt.2008.67. Epub 2008 Apr 8.

12.

Stem cell collection and gene transfer in Fanconi anemia.

Kelly PF, Radtke S, von Kalle C, Balcik B, Bohn K, Mueller R, Schuesler T, Haren M, Reeves L, Cancelas JA, Leemhuis T, Harris R, Auerbach AD, Smith FO, Davies SM, Williams DA.

Mol Ther. 2007 Jan;15(1):211-9.

13.

Gene therapy for fanconi anemia.

Croop JM.

Curr Hematol Rep. 2003 Jul;2(4):335-40. Review.

PMID:
12901331
14.

In vivo repopulation ability of genetically corrected bone marrow cells from Fanconi anemia patients.

Cohen-Haguenauer O, Péault B, Bauche C, Daniel MT, Casal I, Levy V, Dausset J, Boiron M, Auclair C, Gluckman E, Marty M.

Proc Natl Acad Sci U S A. 2006 Feb 14;103(7):2340-5. Epub 2006 Feb 6.

15.

A protocol describing the genetic correction of somatic human cells and subsequent generation of iPS cells.

Raya A, Rodríguez-Pizà I, Navarro S, Richaud-Patin Y, Guenechea G, Sánchez-Danés A, Consiglio A, Bueren J, Izpisúa Belmonte JC.

Nat Protoc. 2010 Apr;5(4):647-60. doi: 10.1038/nprot.2010.9. Epub 2010 Mar 11.

PMID:
20224565
16.

Heterogeneous activation of the Fanconi anemia pathway by patient-derived FANCA mutants.

Adachi D, Oda T, Yagasaki H, Nakasato K, Taniguchi T, D'Andrea AD, Asano S, Yamashita T.

Hum Mol Genet. 2002 Dec 1;11(25):3125-34.

PMID:
12444097
17.

The maternal side of Fanconi Anemia.

Ruiz S, Fernandez-Capetillo O.

Mol Cell. 2014 Sep 18;55(6):803-4. doi: 10.1016/j.molcel.2014.08.029.

18.

Retroviral mediated gene transfer of the Fanconi anemia complementation group C gene to hematopoietic progenitors of group C patients.

Liu JM, Young NS, Walsh CE, Cottler-Fox M, Carter C, Dunbar C, Barrett AJ, Emmons R.

Hum Gene Ther. 1997 Sep 20;8(14):1715-30.

PMID:
9322874
19.

Impaired synthesis of heme oxygenase-1 in Fanconi anemia cells can be rescued by transfection of Fanconi wild-type cDNA.

Kontou M, Hirsch-Kauffmann M, Schweiger M.

Biol Chem. 2008 Oct;389(10):1327-32. doi: 10.1515/BC.2008.151.

PMID:
18713020
20.

Stem cell gene therapy for fanconi anemia: report from the 1st international Fanconi anemia gene therapy working group meeting.

Tolar J, Adair JE, Antoniou M, Bartholomae CC, Becker PS, Blazar BR, Bueren J, Carroll T, Cavazzana-Calvo M, Clapp DW, Dalgleish R, Galy A, Gaspar HB, Hanenberg H, Von Kalle C, Kiem HP, Lindeman D, Naldini L, Navarro S, Renella R, Rio P, Sevilla J, Schmidt M, Verhoeyen E, Wagner JE, Williams DA, Thrasher AJ.

Mol Ther. 2011 Jul;19(7):1193-8. doi: 10.1038/mt.2011.78. Epub 2011 May 3. Review.

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