2 min readLondonNov 6, 2009 02:36 PM IST
First published on: Nov 6, 2009 at 02:36 PM IST
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.
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.
Share
