British researchers are going to start a gene therapy trial in
London for the fatal disorder Duchene muscular dystrophy (DMD), the
first such trial in the world.
A small group of patients will be injected with an experimental
drug which it is hoped to extend their lives, BBC reported
DMD is caused by a single faulty gene on the X-chromosome and
results in progressive muscle wasting. The injection contains a "
molecular patch" targeting the faulty gene so that it should work
The treatment involves scientists making a very small piece of
genetic material -- a molecular patch which once inside the muscle
cell, binds to the piece of code which surrounds the error in the
genetic code on the gene.
The treatment enables the gene to produce a working version of
the dystrophic gene which keeps muscles strong.
Trials of the drug in mice have proved highly successful,
according to researchers.
At first, minute quantities of the drug will be used to check
its safety. If it works the drug will effectively knit together the
key damaged section of DNA, allowing it to begin producing a
protein that keeps the muscles strong.
The researchers said if it works in humans, patients would need
regular infusions of the drug.
Lead researcher Francesco Muntoni from Imperial College London,
has highly hoped that it could slow, or even halt the progression
of muscle wasting, and give some patients the chance of living into
Muntoni describes the gene therapy as like a piece of molecular
velcro which will form a temporary repair.
The trial will be partly funded by the charity Parent Project
UK, the report said.
(Xinhua News Agency October 20, 2007)