Gene therapy for hemophilia

doi:10.1002/1521-2254(200101/02)3:1<3::aid-jgm167>3.0.co;2-h

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Gene therapy for hemophilia

1 Jan 2001

Abstract excerpt

Hemophilia A and B are X-chromosome linked recessive bleeding disorders that result from a deficiency in factor VIII (FVIII) and factor IX (FIX) respectively. Though factor substitution therapy has greatly improved the lives of hemophiliac patients, there are still limitations to the current treatment that have triggered interest in alternative treatments by gene therapy. Significant progress has recently been...

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PMID: 11269333
DOI: 10.1002/1521-2254(200101/02)3:1<3::aid-jgm167>3.0.co;2-h

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