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Gene therapy can cure brain disease: Scientists

In what could raise hopes of thousands of families,scientists have managed to halt a rare brain disease that kills boys by the time of adolescence through an experimental gene therapy.

Written by Agencies | London |
November 6, 2009 2:36:41 pm

In what could raise hopes of thousands of families,scientists have managed to halt a rare brain disease that kills boys by the time of adolescence through an experimental gene therapy.

During the treatment,the team of doctors used a virus known as a lentivirus — a disabled form of HIV — to deliver working genes into two Spanish boys suffering from X-linked drenoleukodystrophy (ALD),a common form of brain disease that affects boys at an early age of six.

“This gives children with ALD and their families the hope of a future,” said Patrick Aubourg,Professor of Paediatrics at Paris Descartes University,who led the research.

“The treatment may also be suitable for adults with a late-onset form of the condition,” he hoped.

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The disease,which was featured in the 1992 academy award-winning movie ‘Lorenzo’s Oil’,affects one boy in 20,000,and is caused by a mutation in the ALD gene,leading to the loss of protective myelin coverings from nerve cells,Times Online reported.

In about 55 per cent of cases,symptoms start between the ages of 6 and 8,causing brain damage and death by the teenage years. The remaining 45 per cent of male carriers develop a less severe form in adulthood,though this can still cause brain damage,paralysis and death.

📣 The above article is for information purposes only and is not intended to be a substitute for professional medical advice. Always seek the guidance of your doctor or other qualified health professional for any questions you may have regarding your health or a medical condition.

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