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RNAi Meets ALS

  March, 22 2005 8:37
your information resource in human molecular genetics
Two reports in the April issue of Nature Medicine show that RNA interference might have therapeutic effects against amyotrophic lateral sclerosis (ALS).

ALS, or Lou Gehrig disease as is often called, is characterized by the death of neurons in the spinal cord, leading to muscle wasting and motor problems. Hereditary forms of the disease are caused by mutations in a protein known as SOD1, and mice engineered to carry a copy of the human gene show symptoms similar to what is seen in people with ALS. Two teams led by Patrick Aebischer and Scott Ralph independently showed that delivering small interfering RNAs that knockdown the synthesis of SOD1 in these mice delays the onset of the disease and slows its progression.

As treatment options for people with ALS are limited, the results of these two groups open a new avenue to pursue in the fight against this debilitating disease.

Author contact:
Patrick Aebischer (Ecole Polytechnique Fédérale de Lausanne, Switzerland)
Tel: + 41 21 693 9521, E-mail: patrick.aebischer@epfl.ch

Scott Ralph (Oxford Biomedica Ltd., United Kingdom)
Tel: + 44 1865 783000, E-mail: s.ralph@oxfordbiomedica.co.uk

Also available online at the following links: Aebischer paper, and Ralph paper.

(C) Nature Medicine press release.

Message posted by: Trevor M. D'Souza

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