UCL  IRIS
Institutional Research Information Service
UCL Logo
Please report any queries concerning the funding data grouped in the sections named "Externally Awarded" or "Internally Disbursed" (shown on the profile page) to your Research Finance Administrator. Your can find your Research Finance Administrator at http://www.ucl.ac.uk/finance/research/post_award/post_award_contacts.php by entering your department
Please report any queries concerning the student data shown on the profile page to:

Email: portico-services@ucl.ac.uk

Help Desk: http://www.ucl.ac.uk/ras/portico/helpdesk
Publication Detail
Gene Therapy for Hemophilia.
  • Publication Type:
    Journal article
  • Publication Sub Type:
    Review
  • Authors:
    Nathwani AC, Davidoff AM, Tuddenham EGD
  • Publication date:
    01/10/2017
  • Pagination:
    853, 868
  • Journal:
    Hematology/oncology clinics of North America
  • Volume:
    31
  • Issue:
    5
  • Medium:
    Print
  • Print ISSN:
    0889-8588
  • Language:
    eng
  • Addresses:
    Department of Academic Haematology, UCL Cancer Institute, Katharine Dormandy Haemophilia and Thrombosis Centre, Rowland Hill Street, London NW3 2PF, United Kingdom; National Health Service Blood and Transplant, Oak House, Reeds Crescent, Watford, Hertfordshire, WD24 4QN, United Kingdom. Electronic address: amit.nathwani@ucl.ac.uk.
Abstract
The best currently available treatments for hemophilia A and B (factor VIII or factor IX deficiency, respectively) require frequent intravenous infusion of highly expensive proteins that have short half-lives. Factor levels follow a saw-tooth pattern that is seldom in the normal range and falls so low that breakthrough bleeding occurs. Most hemophiliacs worldwide do not have access to even this level of care. In stark contrast, gene therapy holds out the hope of a cure by inducing continuous endogenous expression of factor VIII or factor IX following transfer of a functional gene to replace the hemophilic patient's own defective gene.
Publication data is maintained in RPS. Visit https://rps.ucl.ac.uk
 More search options
UCL Researchers
Author
Research Department of Haematology
Author
Research Department of Haematology
University College London - Gower Street - London - WC1E 6BT Tel:+44 (0)20 7679 2000

© UCL 1999–2011

Search by