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Will gene therapy trump factor treatment in hemophilia?

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
1747-4086
43-48
Rangarajan, Savita
9a5e4c7e-55ba-4a3a-b5f6-f1e269d927c3
Aledort, Louis
c4c83796-bf1a-4be4-bf55-6318da91bb7b
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), 43-48. (doi:10.1586/ehm.12.70).

Record type: Review

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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More information

Published date: 1 February 2013
Keywords: ex vivo gene therapy, gene therapy, hemophilia, inhibitors, long-acting factor VIII/IX

Identifiers

Local EPrints ID: 442898
URI: http://eprints.soton.ac.uk/id/eprint/442898
ISSN: 1747-4086
PURE UUID: 6f13fa39-627e-43fc-85b2-fd13dfb57e27
ORCID for Savita Rangarajan: ORCID iD orcid.org/0000-0001-7367-133X

Catalogue record

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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