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Revolutionary treatments developed for rare and common diseases

Posted on 28/02/2011

A 3D computer generated image of a gene therapy viral vector

An international team of leading scientists – including Professor George Dickson from Royal Holloway, University of London – have revealed new gene and stem cell therapies that will revolutionise the treatment of many rare and several common acquired diseases for which no current cure exists.

For the past five years, the scientists have been involved in clinical trials for the treatment of diseases such as inherited spinal diseases and blindness, Parkinson disease, and muscular dystrophy.

The new technologies have been pioneered to develop revolutionary approaches using genetic material and harnessing viruses to bring the medical discipline of gene and stem cell therapy from the laboratory bench to the hospital bedside.

These powerful medical technologies bring with them new and complex dilemmas in medical ethics and medicines regulation. The ‘Clinigene’ network, which the scientists belong to, has organised stakeholder and public consultations and meetings to ensure ethical and regulatory scrutiny of new gene therapies are adequate and uniformly applied across the EU.

Professor Dickson says: “For many severe and often incurable inherited diseases, collaboration between scientists and doctors across the world is key to discover and test new successful gene therapies. Patients are alive today who would otherwise not have survived, and Europe has taken the lead in this area with the CliniGene Network playing a vital role.”

Clinigene is a top EC-funded European academic/industry network (EC-funded) and Royal Holloway is one of two UK partners.

Professor Dickson will be discussing these developments on 28 February at Royal Holloway to mark Rare Disease Day: http://www.rhul.ac.uk/rarediseaseday/home.asp


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