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Gene therapy: progress and predictions
The first clinical gene delivery, which involved insertion of a marker gene into lymphocytes from cancer patients, was published 25 years ago. In this review, we describe progress since then in gene therapy. Patients with some inherited single-gene defects can now be treated with their own bone marr...
Autores principales: | , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
The Royal Society
2015
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4707739/ https://www.ncbi.nlm.nih.gov/pubmed/26702034 http://dx.doi.org/10.1098/rspb.2014.3003 |
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author | Collins, Mary Thrasher, Adrian |
author_facet | Collins, Mary Thrasher, Adrian |
author_sort | Collins, Mary |
collection | PubMed |
description | The first clinical gene delivery, which involved insertion of a marker gene into lymphocytes from cancer patients, was published 25 years ago. In this review, we describe progress since then in gene therapy. Patients with some inherited single-gene defects can now be treated with their own bone marrow stem cells that have been engineered with a viral vector carrying the missing gene. Patients with inherited retinopathies and haemophilia B can also be treated by local or systemic injection of viral vectors. There are also a number of promising gene therapy approaches for cancer and infectious disease. We predict that the next 25 years will see improvements in safety, efficacy and manufacture of gene delivery vectors and introduction of gene-editing technologies to the clinic. Gene delivery may also prove a cost-effective method for the delivery of biological medicines. |
format | Online Article Text |
id | pubmed-4707739 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2015 |
publisher | The Royal Society |
record_format | MEDLINE/PubMed |
spelling | pubmed-47077392016-01-26 Gene therapy: progress and predictions Collins, Mary Thrasher, Adrian Proc Biol Sci Special Feature The first clinical gene delivery, which involved insertion of a marker gene into lymphocytes from cancer patients, was published 25 years ago. In this review, we describe progress since then in gene therapy. Patients with some inherited single-gene defects can now be treated with their own bone marrow stem cells that have been engineered with a viral vector carrying the missing gene. Patients with inherited retinopathies and haemophilia B can also be treated by local or systemic injection of viral vectors. There are also a number of promising gene therapy approaches for cancer and infectious disease. We predict that the next 25 years will see improvements in safety, efficacy and manufacture of gene delivery vectors and introduction of gene-editing technologies to the clinic. Gene delivery may also prove a cost-effective method for the delivery of biological medicines. The Royal Society 2015-12-22 /pmc/articles/PMC4707739/ /pubmed/26702034 http://dx.doi.org/10.1098/rspb.2014.3003 Text en © 2015 The Authors. http://creativecommons.org/licenses/by/4.0/ © 2015 The Authors. Published by the Royal Society under the terms of the Creative Commons Attribution License http://creativecommons.org/licenses/by/4.0/, which permits unrestricted use, provided the original author and source are credited. |
spellingShingle | Special Feature Collins, Mary Thrasher, Adrian Gene therapy: progress and predictions |
title | Gene therapy: progress and predictions |
title_full | Gene therapy: progress and predictions |
title_fullStr | Gene therapy: progress and predictions |
title_full_unstemmed | Gene therapy: progress and predictions |
title_short | Gene therapy: progress and predictions |
title_sort | gene therapy: progress and predictions |
topic | Special Feature |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4707739/ https://www.ncbi.nlm.nih.gov/pubmed/26702034 http://dx.doi.org/10.1098/rspb.2014.3003 |
work_keys_str_mv | AT collinsmary genetherapyprogressandpredictions AT thrasheradrian genetherapyprogressandpredictions |