Imperial College London
Alternate

ProfessorUtaGriesenbach

Faculty of Medicine, National Heart & Lung Institute

Professor of Molecular Medicine
 
 
 
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Contact

 

+44 (0)20 7594 7927u.griesenbach

 
 
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Assistant

 

Miss Samia Soussi +44 (0)20 7594 7980

 
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Location

 

Emmanuel Kaye BuildingRoyal Brompton Campus

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Summary

 

Publications

Citation

BibTex format

@article{Griesenbach:2016:10.1097/MCP.0000000000000327,
author = {Griesenbach, U and Davies, JC and Alton, E},
doi = {10.1097/MCP.0000000000000327},
journal = {Current Opinion in Pulmonary Medicine},
pages = {602--609},
title = {Cystic fibrosis gene therapy: a mutation-independent treatment},
url = {http://dx.doi.org/10.1097/MCP.0000000000000327},
volume = {22},
year = {2016}
}
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RIS format (EndNote, RefMan)

TY  - JOUR
AB  - PURPOSE OF THE REVIEW: Since cloning of the disease-causing gene 27 years ago, the development of cystic fibrosis (CF) gene therapy has been pursued. Here, we will summarize key findings with a particular focus on recent developments. RECENT FINDINGS: Almost 3 decades of research have highlighted the complexity of lung gene transfer and have generated a body of data that has recently led to the completion of a large phase IIB study. This trial has, for the first time, shown that nonviral gene transfer can, albeit modestly, stabilize lung function in CF and provides the impetus for further development of more potent gene transfer agents. Lentiviral vectors, specifically pseudotyped to enable entry into airway epithelial cells have most recently been developed. Persistent expression after a single dose and the ability to be administered repeatedly suggest that these viral vectors hold promise for the treatment of CF; a first-in-man clinical trial will shortly be initiated. SUMMARY: Although the development of CF gene therapy has been slower than initially anticipated, recent progress has been encouraging and has renewed the interest of academics and industry to pursue lung gene therapy.
AU  - Griesenbach,U
AU  - Davies,JC
AU  - Alton,E
DO  - 10.1097/MCP.0000000000000327
EP  - 609
PY  - 2016///
SN  - 1531-6971
SP  - 602
TI  - Cystic fibrosis gene therapy: a mutation-independent treatment
T2  - Current Opinion in Pulmonary Medicine
UR  - http://dx.doi.org/10.1097/MCP.0000000000000327
UR  - http://hdl.handle.net/10044/1/41086
VL  - 22
ER  -
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