Gene Therapy

Our Biomek NX is used to purify RNA/DNA from samples and also to setup a range of assays in the lab. The precision of the 'robot' is greater than human pipetting and allows us to massively increase the throughput of sample processing.

Many diseases such as cystic fibrosis, muscular dystrophy, haemophilia and cancer are caused by faulty genes.

Gene therapy involves the addition of a healthy, working copy of the gene into the appropriate cells of the body to replace or override the faulty copy present in the genome.

So instead of treating the symptoms of the disease, like most conventional medicines, gene therapy has the potential to correct the underlying cause.


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



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




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D.Phil Theses



Gene Therapy Seminars


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