Depakote affects genes

Welcome to the Coping With Epilepsy Forums

Welcome to the Coping With Epilepsy forums - a peer support community for folks dealing (directly or indirectly) with seizure disorders. You can visit the forum page to see the list of forum nodes (categories/rooms) for topics.

Please have a look around and if you like what you see, please consider registering an account and joining the discussions. When you register an account and log in, you may enjoy additional benefits including no ads, access to members only (ie. private) forum nodes and more. Registering an account is free - you have nothing to lose!

Bernard

Your Host
Administrator
Benefactor
Messages
7,392
Reaction score
741
Points
278
This was an interesting article discussing a possible alternative to gene therapy (replacing a faulty gene) to treat neurodegenerative conditions:

...

Another study involving valproate will be presented by Professor Aurora Pujol, of the Medical and Molecular Genetics Centre- IDIBELL and the Institució Catalana de Recerca i Estudis Avançats (the Catalan Research Agency) in Barcelona, Spain. Dr. Pujol and her team are trying to develop new therapies for X-linked adrenoleukodystrophy (X-ALD). This disease involves a single gene on the X chromosome (the ALD gene) and is the most common inherited single-gene disease involving damage to the myelin sheath, which insulates nerve cells in the brain.

Using a mouse model of the disease, Dr Pujol and colleagues found that a protein called ALDR (very similar to the ALD protein mutated in the disease) could, if over-expressed, compensate for the loss of the ALD protein and prevent the development of neurodegenerative symptoms. This striking prevention of the disease lasted during the whole 2 year life span of the mouse.

"Knowing 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."

...

'Smart' Genetic Therapy Helps The Body To Heal Itself
 
Back
Top Bottom