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Royal Free Team Awarded £47,000 From the MRC For Research Into Type II Gene Therapy

In the March 2010 edition of Gauchers News, we reported on the work of Simon Waddington and his team from the Department of Haematology at the Royal Free Hospital, London,which had some positive outcomes.

In 2009 the Association gave a small grant to the team to purchase some viral vectors for use in gene therapy research in Type II Gaucher disease. Simon provides an update on this work which has gone from strength to strength:

On purchasing the vectors we injected these into foetal and neonatal mice and found that we could deliver a marker gene very efficiently to the central and peripheral nervous system. This is an important finding because in Type II - neuronopathic - Gaucher disease the damage to the nervous systemis impossible to treat using enzyme replacement therapy.

I am delighted to report that based on preliminary results, we applied to the UK Medical Research Council to do more extensive studies, initially with marker genes, but then using a gene therapy vector for glucocerebrosidase to treat a mouse model of Type II Gaucher disease which was kindly given to us by Stefan Karlsson from Lund in Sweden.

A month ago we learned that the Medical Research Council has awarded us a grant of £470,000 to perform this work. This will pay for Dr. Ahad Rahim to concentrate his activities on gene therapy for Type II Gaucher disease for three years.We are extremely excited to be embarking upon these studies, particularly given the fast pace at which preclinical and clinical gene therapy is moving. For example, earlier this year, an incurable mousemodel of the disease spinalmuscular atrophy was completely cured by neonatal injection of a similar gene therapy vector which we are planning to use.

We are extremely grateful to the Gauchers Association and the family of Ellie Carter who died from Type II Gaucher disease who initially supported our work through a grant to purchase the initial vectors