Sickle cell disease: testing for cutting-edge techniques | Action Medical Research

Touching Lives - October 2009

Sickle cell disease: testing for cutting-edge techniques

A cutting-edge technique called RNAi could prove useful as part of a new treatment for sickle cell disease.

Sickle cell disease is estimated to affect over 12,000 people in the UK – 20 million worldwide.

Symptoms usually start early, in babies who are just six to nine months old. They face a lifetime of agonisingly painful attacks, known as crises, and possible complications, including strokes, blindness and organ failure. They are also prone to severe infections and periods of anaemia. Sadly, they are not expected to live beyond their 40s or 50s, and some die in childhood.

Perhaps the most successful drug treatment for sickle cell disease is hydroxyurea, but this is only partially effective in most people and ineffectual in others. It seems to work by somehow keeping the gene for a protein called gamma globin switched on, though it kills many cells in the process, meaning it often causes unpleasant side effects.

Researchers at Cranfield University, led by Dr David Carter, are testing whether a technique called RNAi could be a better way to switch the gamma globin gene on. If RNAi works, it could be developed as a new treatment for people with sickle cell disease.

Thanks go to the Esther Appeal Foundation and The Henry Lumley Charitable Trust for part-funding this project.

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