Gene therapy first for children with 'Lorenzo's Oil' disorder

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http://ca.news./s/afp/071029/health/health_disease_children_biotech

Mon Oct 29, 12:57 PM

PARIS (AFP) - Doctors have for the first time successfully used gene

therapy to treat children with a rare disease of the nervous system

that gained worldwide recognition thanks to the Hollywood movie

" Lorenzo's Oil, " a European health group said on Monday.

The European Association for Leukodystrophy (ELA) said the

breakthrough was unveiled by French researchers at a congress in

Rotterdam, the Netherlands, of the European Society of Gene and Cell

Therapy.

The therapy targeted faulty genes that cause adrenoloeukodystrophy

(ALD), one of a group of inherited disorders known as leukodystrophies

in which the protective myeline sheath protecting nerve cells in the

brain becomes damaged.

The disease, transmitted through the mother, affects little boys,

initially affecting their speech, coordination and socialising, and

leads to progressive dementia and death, usually within a decade after

the onset of symptoms.

A team led by Aubourg, a professor at the Saint de

Hospital in Paris, used a harmless version of the AIDS virus as a

Trojan horse, introducing the corrected gene to fix the flawed

chromosome in bone marrow taken from the children.

The bone marrow was then reinjected into the patients.

ALD occurs because a flawed gene on the child's X chromosome does not

produce a key enzyme to break down an accumulation of so-called very

long chain fatty acids in the brain.

Six months after the therapy, the results have been encouraging,

showing a pickup in production of this enzyme, ELA said.

" Lorenzo's Oil, " a 1992 drama starring Nick Nolte and Sarendon,

shows the dogged bid of two parents to devise an oil with fatty acids

that can block myelin destruction.