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Results: 1 to 20 of 112

1.

The gene therapy journey for hemophilia: are we there yet?

High KA.

Hematology Am Soc Hematol Educ Program. 2012;2012:375-81. doi: 10.1182/asheducation-2012.1.375. Review.

PMID:
23233607
[PubMed - indexed for MEDLINE]
Free Article
2.

The gene therapy journey for hemophilia: are we there yet?

High KA.

Blood. 2012 Nov 29;120(23):4482-7. doi: 10.1182/blood-2012-05-423210. Epub 2012 Jul 24. Review.

PMID:
22829631
[PubMed - indexed for MEDLINE]
Free PMC Article
3.

Theodore E. Woodward Award. AAV-mediated gene transfer for hemophilia.

High KA.

Trans Am Clin Climatol Assoc. 2003;114:337-51; discussion 351-2. Review.

PMID:
12813929
[PubMed - indexed for MEDLINE]
Free PMC Article
4.

AAV-mediated gene transfer for hemophilia.

High KA.

Ann N Y Acad Sci. 2001 Dec;953:64-74. Review.

PMID:
11795424
[PubMed - indexed for MEDLINE]
5.

Gene therapy for the hemophilias.

VandenDriessche T, Collen D, Chuah MK.

J Thromb Haemost. 2003 Jul;1(7):1550-8. Review.

PMID:
12871290
[PubMed - indexed for MEDLINE]
6.

AAV-mediated gene therapy for hemophilia.

Couto LB, Pierce GF.

Curr Opin Mol Ther. 2003 Oct;5(5):517-23. Review.

PMID:
14601521
[PubMed - indexed for MEDLINE]
7.

Adeno-associated virus-mediated gene transfer for hemophilia B.

High KA.

Int J Hematol. 2002 Nov;76(4):310-8. Review.

PMID:
12463593
[PubMed - indexed for MEDLINE]
8.

Gene therapy for hemophilia: advancing beyond the first clinical success.

Monahan PE, Gui T.

Curr Opin Hematol. 2013 Sep;20(5):410-6. doi: 10.1097/MOH.0b013e328363c1a1. Review.

PMID:
23852185
[PubMed - indexed for MEDLINE]
9.

Preclinical gene therapy studies for hemophilia using adeno-associated virus (AAV) vectors.

Couto LB.

Semin Thromb Hemost. 2004 Apr;30(2):161-71. Review.

PMID:
15118928
[PubMed - indexed for MEDLINE]
10.

Viral vector-mediated gene therapy for hemophilia.

VandenDriessche T, Collen D, Chuah MK.

Curr Gene Ther. 2001 Sep;1(3):301-15. Review.

PMID:
12109144
[PubMed - indexed for MEDLINE]
11.

Gene therapy for hemophilia.

Lynch CM.

Curr Opin Mol Ther. 1999 Aug;1(4):493-9. Review.

PMID:
11713765
[PubMed - indexed for MEDLINE]
12.

AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B.

Manno CS, Chew AJ, Hutchison S, Larson PJ, Herzog RW, Arruda VR, Tai SJ, Ragni MV, Thompson A, Ozelo M, Couto LB, Leonard DG, Johnson FA, McClelland A, Scallan C, Skarsgard E, Flake AW, Kay MA, High KA, Glader B.

Blood. 2003 Apr 15;101(8):2963-72. Epub 2002 Dec 19.

PMID:
12515715
[PubMed - indexed for MEDLINE]
Free Article
13.

Long-term correction of inhibitor-prone hemophilia B dogs treated with liver-directed AAV2-mediated factor IX gene therapy.

Niemeyer GP, Herzog RW, Mount J, Arruda VR, Tillson DM, Hathcock J, van Ginkel FW, High KA, Lothrop CD Jr.

Blood. 2009 Jan 22;113(4):797-806. doi: 10.1182/blood-2008-10-181479. Epub 2008 Oct 28.

PMID:
18957684
[PubMed - indexed for MEDLINE]
Free PMC Article
14.

Tolerance induction by viral in vivo gene transfer.

Dobrzynski E, Herzog RW.

Clin Med Res. 2005 Nov;3(4):234-40. Review.

PMID:
16303889
[PubMed - indexed for MEDLINE]
Free PMC Article
15.

Efficacy and safety of adeno-associated viral vectors based on serotype 8 and 9 vs. lentiviral vectors for hemophilia B gene therapy.

Vandendriessche T, Thorrez L, Acosta-Sanchez A, Petrus I, Wang L, Ma L, DE Waele L, Iwasaki Y, Gillijns V, Wilson JM, Collen D, Chuah MK.

J Thromb Haemost. 2007 Jan;5(1):16-24. Epub 2006 Sep 26.

PMID:
17002653
[PubMed - indexed for MEDLINE]
16.

Gene therapy for hemophilia "A" and "B": efficacy, safety and immune consequences.

Chuah M, Vandendriessche T.

Verh K Acad Geneeskd Belg. 2007;69(5-6):315-34. Review.

PMID:
18351211
[PubMed - indexed for MEDLINE]
17.

Gene therapy for haemophilia: prospects and challenges to prevent or reverse inhibitor formation.

Scott DW, Lozier JN.

Br J Haematol. 2012 Feb;156(3):295-302. doi: 10.1111/j.1365-2141.2011.08925.x. Epub 2011 Nov 7. Review.

PMID:
22055221
[PubMed - indexed for MEDLINE]
Free PMC Article
18.

Factor IX expression in skeletal muscle of a severe hemophilia B patient 10 years after AAV-mediated gene transfer.

Buchlis G, Podsakoff GM, Radu A, Hawk SM, Flake AW, Mingozzi F, High KA.

Blood. 2012 Mar 29;119(13):3038-41. doi: 10.1182/blood-2011-09-382317. Epub 2012 Jan 23. Erratum in: Blood. 2014 Mar 13;123(11):1768.

PMID:
22271447
[PubMed - indexed for MEDLINE]
Free PMC Article
19.

Gene therapy for hemophilia: clinical trials and technical tribulations.

Viiala NO, Larsen SR, Rasko JE.

Semin Thromb Hemost. 2009 Feb;35(1):81-92. doi: 10.1055/s-0029-1214151. Epub 2009 Mar 23. Review.

PMID:
19308896
[PubMed - indexed for MEDLINE]
20.

Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response.

Manno CS, Pierce GF, Arruda VR, Glader B, Ragni M, Rasko JJ, Ozelo MC, Hoots K, Blatt P, Konkle B, Dake M, Kaye R, Razavi M, Zajko A, Zehnder J, Rustagi PK, Nakai H, Chew A, Leonard D, Wright JF, Lessard RR, Sommer JM, Tigges M, Sabatino D, Luk A, Jiang H, Mingozzi F, Couto L, Ertl HC, High KA, Kay MA.

Nat Med. 2006 Mar;12(3):342-7. Epub 2006 Feb 12. Erratum in: Nat Med. 2006 May;12(5):592. Rasko, John [corrected to Rasko, John JE]; Rustagi, Pradip K [added].

PMID:
16474400
[PubMed - indexed for MEDLINE]

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