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Gene treatment's new promise

Tuesday July 26th, 2011

A much-heralded new treatment for a once incurable genetic disease continues to show promise, British researchers have reported.

Some 19 boys aged five to 15 with muscular dystrophy have been testing the "exon skipping" gene treatment in London and Newcastle, UK.

The latest report, published yesterday in the Lancet, shows encouraging signs in seven of the boys.

Duchenne muscular dystrophy was identified early as a disease that could be treated by gene therapy after the discovery of its genetic causes - but attempts to develop a treatment have been slow to show success.

The latest treatment aims to restore a missing protein known as dystrophin in the muscles.

The study showed that in the seven boys protein levels reached 18 per cent of normal levels after 12 weeks of treatment with the new drug, known as AVI-4658, which was developed in Australia.

Researcher Professor Francesco Muntoni, of the UCL Institute of Child Health, London, says the findings show the treatment could have a "positive effect on the prevention of muscle degeneration in Duchenne muscular dystrophy."

Writing in the journal, Japanese experts says the new treatment may also be simpler to implement than other kinds of gene therapy.

Dr Akinori Nakamura, of the Shinshu University School of Medicine, Matsumoto, and Dr Shin'ichi Takeda, of the National Institute of Neuroscience, National Centre of Neurology and Psychiatry, Tokyo, say: "The regulatory and practical (ie, scaling up) barriers to clinical use of exon-skipping therapy are much less daunting than those for gene therapy with viral vectors or cell transplantation therapy.

"Therefore, successful clinical trials of exon skipping therapy in patients with Duchenne muscular dystrophy could have a great effect on development of treatments for other intractable hereditary neuromuscular disorders."

The Lancet July 25 2011

Tags: Asia | Child Health | Genetics | UK News

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