Cargando…

Taking stock of gene therapy for cystic fibrosis

The identification of the cystic fibrosis (CF) gene opened the way for gene therapy. In the ten years since then, proof of principle in vitro and then in animal models in vivo has been followed by numerous clinical studies using both viral and non-viral vectors to transfer normal copies of the gene...

Descripción completa

Detalles Bibliográficos
Autores principales: Stern, Myra, Geddes, Duncan M, Alton, Eric WFW
Formato: Texto
Lenguaje:English
Publicado: BioMed Central 2000
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC59546/
https://www.ncbi.nlm.nih.gov/pubmed/11667969
http://dx.doi.org/10.1186/rr16
_version_ 1782120069604573184
author Stern, Myra
Geddes, Duncan M
Alton, Eric WFW
author_facet Stern, Myra
Geddes, Duncan M
Alton, Eric WFW
author_sort Stern, Myra
collection PubMed
description The identification of the cystic fibrosis (CF) gene opened the way for gene therapy. In the ten years since then, proof of principle in vitro and then in animal models in vivo has been followed by numerous clinical studies using both viral and non-viral vectors to transfer normal copies of the gene to the lungs and noses of CF patients. A wealth of data have emerged from these studies, reflecting enormous progress and also helping to focus and define key difficulties that remain unresolved. Gene therapy for CF remains the most promising possibility for curative rather than symptomatic therapy.
format Text
id pubmed-59546
institution National Center for Biotechnology Information
language English
publishDate 2000
publisher BioMed Central
record_format MEDLINE/PubMed
spelling pubmed-595462001-11-06 Taking stock of gene therapy for cystic fibrosis Stern, Myra Geddes, Duncan M Alton, Eric WFW Respir Res Commentary The identification of the cystic fibrosis (CF) gene opened the way for gene therapy. In the ten years since then, proof of principle in vitro and then in animal models in vivo has been followed by numerous clinical studies using both viral and non-viral vectors to transfer normal copies of the gene to the lungs and noses of CF patients. A wealth of data have emerged from these studies, reflecting enormous progress and also helping to focus and define key difficulties that remain unresolved. Gene therapy for CF remains the most promising possibility for curative rather than symptomatic therapy. BioMed Central 2000 2000-09-15 /pmc/articles/PMC59546/ /pubmed/11667969 http://dx.doi.org/10.1186/rr16 Text en Copyright © 2000 Current Science Ltd
spellingShingle Commentary
Stern, Myra
Geddes, Duncan M
Alton, Eric WFW
Taking stock of gene therapy for cystic fibrosis
title Taking stock of gene therapy for cystic fibrosis
title_full Taking stock of gene therapy for cystic fibrosis
title_fullStr Taking stock of gene therapy for cystic fibrosis
title_full_unstemmed Taking stock of gene therapy for cystic fibrosis
title_short Taking stock of gene therapy for cystic fibrosis
title_sort taking stock of gene therapy for cystic fibrosis
topic Commentary
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC59546/
https://www.ncbi.nlm.nih.gov/pubmed/11667969
http://dx.doi.org/10.1186/rr16
work_keys_str_mv AT sternmyra takingstockofgenetherapyforcysticfibrosis
AT geddesduncanm takingstockofgenetherapyforcysticfibrosis
AT altonericwfw takingstockofgenetherapyforcysticfibrosis