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7 July 2003.
Scientists with funding from the Muscular Dystrophy
Campaign (and others) have reported encouraging results in
their search for effective treatments for Duchenne muscular
dystrophy (DMD).
DMD is a fatal condition which causes muscle wasting and
weakening and affects 1 in 3500 boys in the UK. It arises
from genetic errors on the X chromosome which disrupt the
production of a protein called dystrophin. Without
dystrophin the membrane of the muscle cell is weakened and
the cell eventually dies. Mutations in the code's sequence
can make it "unreadable", resulting in an absence of
functional dystrophin.
Now, an article in
Nature
Medicine, describes research using a "molecular patch"
to cover the genetic error, altering the gene's sequencing
for therapeutic effect. Earlier research using this
technique in cultured DMD skin cells has demonstrated its
potential to produce dystrophin in muscle cells. However,
attempts to introduce "molecular patches" into mice were far
less effective and only resulted in limited, localised
effect.
The report in Nature Medicine by Professor Terence
Partridge and colleagues reveals that an important next step
has been taken. The researchers were able to deliver
sufficient quantities of the "molecular patch" into mice leg
muscles, using a substance, F127, that had previously been
shown to enhance delivery of proteins into cells.
Encouragingly, significant levels of functional dystrophin
were produced in the muscle that was injected, and the
dystrophin remained at relatively high levels for several
months. Nor was there any sign of an immune reaction, even
on repeated administration, which is essential because this
type of therapy would require constant levels of dystrophin.
"This research is very encouraging. We are getting nearer
to finding a way to deliver a molecular patch so that
genetic mutations no longer prevent the production of the
essential muscle protein, dystrophin. Further research into
delivery systems that target all affected muscles is
desperately needed" said Jenny Versnel, Head of research at
the Muscular Dystrophy Campaign.
Joe Korner, Director of Corporate Affairs at the Muscular
Dystrophy Campaign said "Significant advances, such as this
research, serve to show how much progress has been made in
the search for treatments for DMD, but also how urgently we
need more funds for research. The Government's White paper
on Genetics, published last month, provides a welcome boost
to funding for gene therapy research but we need to invest
even more if we are take the next steps in finding effective
treatments and eventually cures for DMD and other muscle
wasting disorders. That's why the Muscular Dystrophy
Campaign is calling on the Government to commit £20 million
over 5 years into muscular dystrophy research."
See BBC Online
news report.
Contact: Joe Korner, Muscular Dystrophy Campaign 020 7819
8182 |
