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'Smart' genetic therapy helps the body to heal itself

nowing that a class of drug called histone deacetilase (HDAC) inhibitors was able to stimulate the expression of ALDR in the mouse and in human cell lines, we became interested by the work of Professor Wirth's laboratory on valproate, which belongs to the HDAC class and enters the brain ", said Professor Pujol.

The scientists tested valproate and found that it could indeed induce ALDR gene expression in human cell lines and rat and human brain slices. "We then treated 8 X-ALD patients with oral valproate over 6 months", said Professor Pujol. "We collected samples before treatment, and at 3 and 6 months, and on analysing the amount of ALDR in their white blood cells, we found that half of them showed increased levels."

The scientists now intend to measure the actual marker of the disease, the very-long chain fatty acids in white blood cells and plasma. If these levels have normalised with valproate, they hope to be able to start a double-blind clinical trial in collaboration with Professor Patrick Aubourg of INSERM, the French national medical research agency, at the Hospital Saint Vincent de Paul, Paris.

"Treatment with valproate could ameliorate symptoms or prevent neurodegeneration, especially in the late-onset type of the disease", said Professor Pujol. "Currently there is no effective therapy available for this devastating disease, which can cause visual loss, seizures, speech problems or deafness due to demyelination invariably leading to death in children and problems of gait and co-ordination in adults."

Professor Gert-Jan van Ommen, from the Centre for Human and Clinical Genetics, Leiden University Medical Centre, The Netherlands, and colleagues, will tell the conference that the team of Dr. Judith van Deutekom in his department has discovered a promising genetic therapy for Duchenne muscular dystrophy (DMD). Using anti-sense molecules (small pieces of synthetic RNA that can bind to specific sequences in the parts
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Source:European Society of Human Genetics


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