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How will the field of gene therapy survive its success?

In August 2017, for the first time, a gene therapy was approved for market release in the United States. That approval was followed by two others before the end of the year. This article cites primary literature, review articles concerning particular biotechnologies, and press releases by the FDA an...

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Autor principal: Kaemmerer, William F.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley and Sons Inc. 2018
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6063870/
https://www.ncbi.nlm.nih.gov/pubmed/30065971
http://dx.doi.org/10.1002/btm2.10090
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author Kaemmerer, William F.
author_facet Kaemmerer, William F.
author_sort Kaemmerer, William F.
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description In August 2017, for the first time, a gene therapy was approved for market release in the United States. That approval was followed by two others before the end of the year. This article cites primary literature, review articles concerning particular biotechnologies, and press releases by the FDA and others in order to provide an overview of the current status of the field of gene therapy with respect to its translation into practice. Technical hurdles that have been overcome in the past decades are summarized, as are hurdles that need to be the subject of continued research. Then, some social and practical challenges are identified that must be overcome if the field of gene therapy, having survived past failures, is to achieve not only technical and clinical but also market success. One of these, the need for an expanded capacity for the manufacturing of viral vectors to be able to meet the needs of additional gene therapies that will be coming soon, is a challenge that the talents of current and future bioengineers may help address.
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spelling pubmed-60638702018-07-31 How will the field of gene therapy survive its success? Kaemmerer, William F. Bioeng Transl Med Review In August 2017, for the first time, a gene therapy was approved for market release in the United States. That approval was followed by two others before the end of the year. This article cites primary literature, review articles concerning particular biotechnologies, and press releases by the FDA and others in order to provide an overview of the current status of the field of gene therapy with respect to its translation into practice. Technical hurdles that have been overcome in the past decades are summarized, as are hurdles that need to be the subject of continued research. Then, some social and practical challenges are identified that must be overcome if the field of gene therapy, having survived past failures, is to achieve not only technical and clinical but also market success. One of these, the need for an expanded capacity for the manufacturing of viral vectors to be able to meet the needs of additional gene therapies that will be coming soon, is a challenge that the talents of current and future bioengineers may help address. John Wiley and Sons Inc. 2018-05-24 /pmc/articles/PMC6063870/ /pubmed/30065971 http://dx.doi.org/10.1002/btm2.10090 Text en © 2018 The Authors. Bioengineering & Translational Medicine is published by Wiley Periodicals, Inc. on behalf of The American Institute of Chemical Engineers This is an open access article under the terms of the http://creativecommons.org/licenses/by/4.0/ License, which permits use, distribution and reproduction in any medium, provided the original work is properly cited.
spellingShingle Review
Kaemmerer, William F.
How will the field of gene therapy survive its success?
title How will the field of gene therapy survive its success?
title_full How will the field of gene therapy survive its success?
title_fullStr How will the field of gene therapy survive its success?
title_full_unstemmed How will the field of gene therapy survive its success?
title_short How will the field of gene therapy survive its success?
title_sort how will the field of gene therapy survive its success?
topic Review
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6063870/
https://www.ncbi.nlm.nih.gov/pubmed/30065971
http://dx.doi.org/10.1002/btm2.10090
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