Turning genes into medicines—what have we learned from gene therapy drug development in the past decade?

Gene and cell therapy products approved over the past decade in Europe and North America have provided new therapeutic options for single gene disorders and for hematologic malignancies. Lessons learned, and limitations identified, are reviewed.

Detalles Bibliográficos
Autor principal: High, Katherine A.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2020
Materias:
Comment
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7669843/
https://www.ncbi.nlm.nih.gov/pubmed/33199678
http://dx.doi.org/10.1038/s41467-020-19507-0
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author High, Katherine A.
author_facet High, Katherine A.
author_sort High, Katherine A.
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description Gene and cell therapy products approved over the past decade in Europe and North America have provided new therapeutic options for single gene disorders and for hematologic malignancies. Lessons learned, and limitations identified, are reviewed.
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spelling pubmed-76698432020-11-24 Turning genes into medicines—what have we learned from gene therapy drug development in the past decade? High, Katherine A. Nat Commun Comment Gene and cell therapy products approved over the past decade in Europe and North America have provided new therapeutic options for single gene disorders and for hematologic malignancies. Lessons learned, and limitations identified, are reviewed. Nature Publishing Group UK 2020-11-16 /pmc/articles/PMC7669843/ /pubmed/33199678 http://dx.doi.org/10.1038/s41467-020-19507-0 Text en © The Author(s) 2020 Open Access This article is licensed under a Creative Commons Attribution 4.0 International License, which permits use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons license, and indicate if changes were made. The images or other third party material in this article are included in the article’s Creative Commons license, unless indicated otherwise in a credit line to the material. If material is not included in the article’s Creative Commons license and your intended use is not permitted by statutory regulation or exceeds the permitted use, you will need to obtain permission directly from the copyright holder. To view a copy of this license, visit http://creativecommons.org/licenses/by/4.0/.
spellingShingle Comment
High, Katherine A.
Turning genes into medicines—what have we learned from gene therapy drug development in the past decade?
title Turning genes into medicines—what have we learned from gene therapy drug development in the past decade?
title_full Turning genes into medicines—what have we learned from gene therapy drug development in the past decade?
title_fullStr Turning genes into medicines—what have we learned from gene therapy drug development in the past decade?
title_full_unstemmed Turning genes into medicines—what have we learned from gene therapy drug development in the past decade?
title_short Turning genes into medicines—what have we learned from gene therapy drug development in the past decade?
title_sort turning genes into medicines—what have we learned from gene therapy drug development in the past decade?
topic Comment
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7669843/
https://www.ncbi.nlm.nih.gov/pubmed/33199678
http://dx.doi.org/10.1038/s41467-020-19507-0
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