Abstract
The limitations of transfusion therapy for haemophilia have prompted efforts to develop gene therapy for these disorders. The coagulation factors have rather short half-lives, and cannot be adequately replaced to prevent all episodes of bleeding in haemophiliacs [1]. Multiple transfusions are required at times of major trauma or surgery. The benefit of gene therapy would be continuous production of the deficient coagulation factor, preferably after a single treatment which would allow permanent factor replacement.
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Koeberl, D.D., Krumm, A., Miller, A.D. (1995). Inhibitory Sequences within the Clotting Factor VIII cDNA Block Transcriptional Elongation and Complicate Efforts Toward Gene Therapy for Haemophilia A. In: Sibinga, C.T.S., Das, P.C., Briët, E. (eds) Hereditary Diseases and Blood Transfusion. Developments in Hematology and Immunology, vol 30. Springer, Boston, MA. https://doi.org/10.1007/978-1-4615-2017-7_4
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DOI: https://doi.org/10.1007/978-1-4615-2017-7_4
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