<abstract xmlns="http://www.w3.org/1999/xhtml"><p> Clotting factor replacement therapy has proven a highly effective means of treating haemophilia A and B. But treatment involves frequent and lifelong infusion of factor concentrates and is generally prophylactic rather than curative. It is also extremely expensive, associated with inhibitor formation and does not fully abolish the potential for spontaneous bleeding. Gene therapy offers a potential cure for haemophilia, with the possible continuous expression of a clotting factor gene following the administration of a viral vector carrying the appropriate gene. Recent clinical trials of gene therapy for haemophilia have proven positive in selected patients and new studies are underway.</p></abstract>

Clotting factor replacement therapy has proven a highly effective means of treating haemophilia A and B. But treatment involves frequent and lifelong infusion of factor concentrates and is generally prophylactic rather than curative. It is also extremely expensive, associated with inhibitor formation and does not fully abolish the potential for spontaneous bleeding. Gene therapy offers a potential cure for haemophilia, with the possible continuous expression of a clotting factor gene following the administration of a viral vector carrying the appropriate gene. Recent clinical trials of gene therapy for haemophilia have proven positive in selected patients and new studies are underway.

Gene therapy for haemophilia: an update on progress in clinical trials

The Journal of Haemophilia Practice

This work is licensed under the Creative Commons Attribution-NonCommercial-NoDerivatives 4.0 License.

{"article-title":"Gene therapy for haemophilia: an update on progress in clinical trials"}

Clotting factor replacement therapy has proven a highly effective means of treating haemophilia A and B. But treatment involves frequent and lifelong infusion...

Gene therapy for haemophilia: an update on progress in clinical trials

Article Category: Gene Therapy

Published Online: Apr 21, 2018

Page range: 3 - 5

DOI: https://doi.org/10.17225/jhp.00039

Keywords
haemophilia, haemophilia B, Christmas disease, gene therapy

© 2018

This work is licensed under the Creative Commons Attribution-NonCommercial-NoDerivatives 4.0 License.

Gene therapy for haemophilia: an update on progress in clinical trials

Article Category: Gene Therapy

Published Online: Apr 21, 2018

Page range: 3 - 5

DOI: https://doi.org/10.17225/jhp.00039

Keywordshaemophilia, haemophilia B, Christmas disease, gene therapy

© 2018

This work is licensed under the Creative Commons Attribution-NonCommercial-NoDerivatives 4.0 License.

Keywords
haemophilia, haemophilia B, Christmas disease, gene therapy