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Gene therapy for haemophilia

7 Feb 2008

Abstract excerpt

The ultimate goal of gene therapy is the replacement of a defective gene sequence with a corrected version to eliminate disease for the lifetime of the patient. This challenging task is not yet accomplished, however significant progress is evident. An initial spate of clinical trials attempting the treatment of haemophilia with gene transfer primarily resulted in the demonstration of good safety profiles, but...

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Identifiers

PMID: 18275425
DOI: 10.1111/j.1365-2141.2007.06942.x

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