Gene therapy for cystic fibrosis: new tools for precision medicine

The discovery of the Cystic fibrosis (CF) gene in 1989 has paved the way for incredible progress in treating the disease such that the mean survival age of individuals living with CF is now ~58 years in Canada. Recent developments in gene targeting tools and new cell and animal models have re-ignite...

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Autores principales: Lee, Jin-A, Cho, Alex, Huang, Elena N., Xu, Yiming, Quach, Henry, Hu, Jim, Wong, Amy P.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2021
Materias:
Review
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8556969/
https://www.ncbi.nlm.nih.gov/pubmed/34717671
http://dx.doi.org/10.1186/s12967-021-03099-4
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author Lee, Jin-A
Cho, Alex
Huang, Elena N.
Xu, Yiming
Quach, Henry
Hu, Jim
Wong, Amy P.
author_facet Lee, Jin-A
Cho, Alex
Huang, Elena N.
Xu, Yiming
Quach, Henry
Hu, Jim
Wong, Amy P.
author_sort Lee, Jin-A
collection PubMed
description The discovery of the Cystic fibrosis (CF) gene in 1989 has paved the way for incredible progress in treating the disease such that the mean survival age of individuals living with CF is now ~58 years in Canada. Recent developments in gene targeting tools and new cell and animal models have re-ignited the search for a permanent genetic cure for all CF. In this review, we highlight some of the more recent gene therapy approaches as well as new models that will provide insight into personalized therapies for CF.
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spelling pubmed-85569692021-11-01 Gene therapy for cystic fibrosis: new tools for precision medicine Lee, Jin-A Cho, Alex Huang, Elena N. Xu, Yiming Quach, Henry Hu, Jim Wong, Amy P. J Transl Med Review The discovery of the Cystic fibrosis (CF) gene in 1989 has paved the way for incredible progress in treating the disease such that the mean survival age of individuals living with CF is now ~58 years in Canada. Recent developments in gene targeting tools and new cell and animal models have re-ignited the search for a permanent genetic cure for all CF. In this review, we highlight some of the more recent gene therapy approaches as well as new models that will provide insight into personalized therapies for CF. BioMed Central 2021-10-30 /pmc/articles/PMC8556969/ /pubmed/34717671 http://dx.doi.org/10.1186/s12967-021-03099-4 Text en © The Author(s) 2021 https://creativecommons.org/licenses/by/4.0/Open AccessThis 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 licence, and indicate if changes were made. The images or other third party material in this article are included in the article's Creative Commons licence, unless indicated otherwise in a credit line to the material. If material is not included in the article's Creative Commons licence 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 licence, visit http://creativecommons.org/licenses/by/4.0/ (https://creativecommons.org/licenses/by/4.0/) . The Creative Commons Public Domain Dedication waiver (http://creativecommons.org/publicdomain/zero/1.0/ (https://creativecommons.org/publicdomain/zero/1.0/) ) applies to the data made available in this article, unless otherwise stated in a credit line to the data.
spellingShingle Review
Lee, Jin-A
Cho, Alex
Huang, Elena N.
Xu, Yiming
Quach, Henry
Hu, Jim
Wong, Amy P.
Gene therapy for cystic fibrosis: new tools for precision medicine
title Gene therapy for cystic fibrosis: new tools for precision medicine
title_full Gene therapy for cystic fibrosis: new tools for precision medicine
title_fullStr Gene therapy for cystic fibrosis: new tools for precision medicine
title_full_unstemmed Gene therapy for cystic fibrosis: new tools for precision medicine
title_short Gene therapy for cystic fibrosis: new tools for precision medicine
title_sort gene therapy for cystic fibrosis: new tools for precision medicine
topic Review
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8556969/
https://www.ncbi.nlm.nih.gov/pubmed/34717671
http://dx.doi.org/10.1186/s12967-021-03099-4
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