Batty, P;
Lillicrap, D;
(2021)
Hemophilia Gene Therapy: Approaching the First Licensed Product.
HemaSphere
, 5
(3)
, Article E540. 10.1097/HS9.0000000000000540.
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Abstract
The clinical potential of hemophilia gene therapy has now been pursued for the past 30 years, and there is a realistic expectation that this goal will be achieved within the next couple of years with the licensing of a gene therapy product. While recent late phase clinical trials of hemophilia gene therapy have shown promising results, there remain a number of issues that require further attention with regard to both efficacy and safety of this therapeutic approach. In this review, we present information relating to the current status of the field and focus attention on the unanswered questions for hemophilia gene therapy and the future challenges that need to be overcome to enable the widespread application of this treatment paradigm.
| Type: | Article |
|---|---|
| Title: | Hemophilia Gene Therapy: Approaching the First Licensed Product |
| Open access status: | An open access version is available from UCL Discovery |
| DOI: | 10.1097/HS9.0000000000000540 |
| Publisher version: | https://doi.org/10.1097/hs9.0000000000000540 |
| Language: | English |
| Additional information: | This work is licensed under a Creative Commons License. The images or other third-party material in this article are included in the Creative Commons license, unless indicated otherwise in the credit line; if the material is not included under the Creative Commons license, users will need to obtain permission from the license holder to reproduce the material. To view a copy of this license, visit http://creativecommons.org/licenses/by/4.0/ |
| UCL classification: | UCL UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences > Cancer Institute UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences > Cancer Institute > Research Department of Haematology |
| URI: | https://discovery.ucl.ac.uk/id/eprint/10207623 |
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