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Items: 16

1.

Adenovirus-associated virus vector-mediated gene transfer in hemophilia B.

Nathwani AC, Tuddenham EG, Rangarajan S, Rosales C, McIntosh J, Linch DC, Chowdary P, Riddell A, Pie AJ, Harrington C, O'Beirne J, Smith K, Pasi J, Glader B, Rustagi P, Ng CY, Kay MA, Zhou J, Spence Y, Morton CL, Allay J, Coleman J, Sleep S, Cunningham JM, Srivastava D, Basner-Tschakarjan E, Mingozzi F, High KA, Gray JT, Reiss UM, Nienhuis AW, Davidoff AM.

N Engl J Med. 2011 Dec 22;365(25):2357-65. doi: 10.1056/NEJMoa1108046.

2.

New and improved AAVenues: current status of hemophilia B gene therapy.

Brimble MA, Reiss UM, Nathwani AC, Davidoff AM.

Expert Opin Biol Ther. 2016;16(1):79-92. doi: 10.1517/14712598.2015.1106475. Review.

PMID:
26524468
3.

AAV-mediated gene transfer for hemophilia.

High KA.

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

PMID:
11795424
4.

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.

5.

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

High KA.

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

PMID:
12463593
6.

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. Review.

7.

AAV-mediated gene transfer for the treatment of hemophilia B: problems and prospects.

Hasbrouck NC, High KA.

Gene Ther. 2008 Jun;15(11):870-5. doi: 10.1038/gt.2008.71. Review.

PMID:
18432276
8.

Translational data from adeno-associated virus-mediated gene therapy of hemophilia B in dogs.

Nichols TC, Whitford MH, Arruda VR, Stedman HH, Kay MA, High KA.

Hum Gene Ther Clin Dev. 2015 Mar;26(1):5-14. doi: 10.1089/humc.2014.153. Review.

9.

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
10.

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
11.

Immune responses to AAV in clinical trials.

Mingozzi F, High KA.

Curr Gene Ther. 2007 Oct;7(5):316-24. Review.

PMID:
17979678
12.

Gene therapy for the hemophilias.

VandenDriessche T, Collen D, Chuah MK.

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

13.

Gene therapy in an era of emerging treatment options for hemophilia B.

Monahan PE.

J Thromb Haemost. 2015 Jun;13 Suppl 1:S151-60. doi: 10.1111/jth.12957. Review.

14.

Technology evaluation: AAV factor IX gene therapy, Avigen Inc.

Fabb SA, Dickson JG.

Curr Opin Mol Ther. 2000 Oct;2(5):601-6. Review.

PMID:
11249763
15.

Progress towards gene therapy for haemophilia B.

Patel N, Reiss U, Davidoff AM, Nathwani AC.

Int J Hematol. 2014 Apr;99(4):372-6. doi: 10.1007/s12185-014-1523-0. Review.

16.

Self-complementary adeno-associated viral vectors for gene therapy of hemophilia B: progress and challenges.

Raj D, Davidoff AM, Nathwani AC.

Expert Rev Hematol. 2011 Oct;4(5):539-49. doi: 10.1586/ehm.11.48. Review.

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