Assessment of CFTR function after gene transfer in vitro and in vivo (2008)

Griesenbach, U., Munkonge, F. M., Sumner-Jones, S., Holder, E., Smith, S. N., Boyd, A. C., Gill, D. R., Hyde, S. C., Porteous, D. & Alton, E. W.

Methods Mol Biol, 433, 229-242

Pubmed   Back

Cystic fibrosis (CF) a monogenic lethal disease and, therefore, ideally suited for the development of gene therapy. The first clinical trials were carried out shortly after cloning the CF gene in 1989. Since then, 25 trials have been carried out. Proof of principle for low-level airway gene transfer was established in most, but not all, trials. It is currently unclear whether current gene transfer efficiency will lead to improvements in clinically relevant endpoints such as inflammation or infection. In addition to addressing this important question, we and others are further improving airway gene transfer, by modifying existing and developing new gene transfer agents. Here, we describe pre-clinical methods related to assessing correction of the CF chloride transport defect.

Home
News
Introductory Videos
Medical Futures Innovation Award 2011
Twitter Feed
About Us
Contact Us
Careers
Resources
Lab Events
Environemental Policy
About this Site

Google Site Search

Site Feedback Form

All Site Images

 

UK CFGTC

How the Consortium works/FAQs

Consortium Website

Centre for Molecular Medicine, Edinburgh
The Roslin Institute
Dep of Gene Therapy, Imperial

Milestones

GL67A/pGM169

The Run-in Study

Single Dose Clinical Trial

Multi Dose Clinical Trial

 

Our Research

Non-viral Vector Development

Aerosol Mediated Gene Delivery

Viral Vector Development

Taqman Core Facility

Cystic Fibrosis

History of CF

Discovery of the CFTR Gene

CFTR Protein Structure

CFTR Function

CF Links

 

Gene Therapy

Introduction to Gene Therapy

Other CF Gene Therapy Groups

Why use Gene Therapy for CF

Target Cells for CF Gene Therapy

Barriers for CF Gene Therapy

Clinical Trials

Gene Therapy Successes

Gene Therapy Links

 

 

Publications

Papers in Journals

Conference Posters & Presentations

Book Chapters

D.Phil Theses

Lectures

 

Gene Therapy Seminars

Schedule

Directions & Venue