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Hemophilia Gene Therapy: Approaching the First Licensed Product
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...
Autores principales: | , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Lippincott Williams & Wilkins
2021
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7886458/ https://www.ncbi.nlm.nih.gov/pubmed/33604517 http://dx.doi.org/10.1097/HS9.0000000000000540 |
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author | Batty, Paul Lillicrap, David |
author_facet | Batty, Paul Lillicrap, David |
author_sort | Batty, Paul |
collection | PubMed |
description | 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. |
format | Online Article Text |
id | pubmed-7886458 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2021 |
publisher | Lippincott Williams & Wilkins |
record_format | MEDLINE/PubMed |
spelling | pubmed-78864582021-02-17 Hemophilia Gene Therapy: Approaching the First Licensed Product Batty, Paul Lillicrap, David Hemasphere Review Article 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. Lippincott Williams & Wilkins 2021-02-10 /pmc/articles/PMC7886458/ /pubmed/33604517 http://dx.doi.org/10.1097/HS9.0000000000000540 Text en Copyright © 2021 the Author(s). Published by Wolters Kluwer Health, Inc. on behalf of the European Hematology Association. This is an open access article distributed under the Creative Commons Attribution License 4.0 (CCBY) (http://creativecommons.org/licenses/by/4.0/) , which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited. |
spellingShingle | Review Article Batty, Paul Lillicrap, David Hemophilia Gene Therapy: Approaching the First Licensed Product |
title | Hemophilia Gene Therapy: Approaching the First Licensed Product |
title_full | Hemophilia Gene Therapy: Approaching the First Licensed Product |
title_fullStr | Hemophilia Gene Therapy: Approaching the First Licensed Product |
title_full_unstemmed | Hemophilia Gene Therapy: Approaching the First Licensed Product |
title_short | Hemophilia Gene Therapy: Approaching the First Licensed Product |
title_sort | hemophilia gene therapy: approaching the first licensed product |
topic | Review Article |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7886458/ https://www.ncbi.nlm.nih.gov/pubmed/33604517 http://dx.doi.org/10.1097/HS9.0000000000000540 |
work_keys_str_mv | AT battypaul hemophiliagenetherapyapproachingthefirstlicensedproduct AT lillicrapdavid hemophiliagenetherapyapproachingthefirstlicensedproduct |