Will gene therapy trump factor treatment in hemophilia?
Will gene therapy trump factor treatment in hemophilia?
Hemophilia treatment is entering a new phase, with the exciting possibility of gene therapy promising a cure. Novel gene transfer strategies are being considered for patients with inhibitors.Improvement of factor-replacement therapy is being aggressively pursued with long-acting factor concentrates, many of which are in clinical trials. Whether gene therapy will be safe and cost effective to eventually supersede factor-replacement therapy is yet to be determined. It is hoped that with the profusion of clinical trial programs in hemophilia care, it will eventually provide affordable treatment to many patients who currently cannot access adequate treatment in the developing countries.
ex vivo gene therapy, gene therapy, hemophilia, inhibitors, long-acting factor VIII/IX
43-48
Rangarajan, Savita
9a5e4c7e-55ba-4a3a-b5f6-f1e269d927c3
Aledort, Louis
c4c83796-bf1a-4be4-bf55-6318da91bb7b
1 February 2013
Rangarajan, Savita
9a5e4c7e-55ba-4a3a-b5f6-f1e269d927c3
Aledort, Louis
c4c83796-bf1a-4be4-bf55-6318da91bb7b
Rangarajan, Savita and Aledort, Louis
(2013)
Will gene therapy trump factor treatment in hemophilia?
Expert Review of Hematology, 6 (1), .
(doi:10.1586/ehm.12.70).
Abstract
Hemophilia treatment is entering a new phase, with the exciting possibility of gene therapy promising a cure. Novel gene transfer strategies are being considered for patients with inhibitors.Improvement of factor-replacement therapy is being aggressively pursued with long-acting factor concentrates, many of which are in clinical trials. Whether gene therapy will be safe and cost effective to eventually supersede factor-replacement therapy is yet to be determined. It is hoped that with the profusion of clinical trial programs in hemophilia care, it will eventually provide affordable treatment to many patients who currently cannot access adequate treatment in the developing countries.
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Published date: 1 February 2013
Keywords:
ex vivo gene therapy, gene therapy, hemophilia, inhibitors, long-acting factor VIII/IX
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Local EPrints ID: 442898
URI: http://eprints.soton.ac.uk/id/eprint/442898
ISSN: 1747-4086
PURE UUID: 6f13fa39-627e-43fc-85b2-fd13dfb57e27
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Date deposited: 30 Jul 2020 16:34
Last modified: 17 Mar 2024 04:02
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Author:
Louis Aledort
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