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Researchers 'grow Rett syndrome' in a Petri dish
Date:11/15/2010

o date, no effective pharmacological treatments have been developed.

The main limitation for human studies and thus, drug development, has been the inaccessibility of live neurons from human patients. To get around this issue, the use of induced pluripotent stem cells (iPSCs) to establish a human cell-based model of Rett syndrome is key to the development of high throughput drug screens and therefore has been an area of the highest priority for IRSF who provided a post-doctoral fellowship Award to Dr. Cassiano Carromeu, a member of the team and a co-author of the current study. The study's principal author, Dr. Alysson Muotri, said, "Dr. Bird's data shows that you can reverse symptoms in a mouse model, now we've shown that this could be done using human cells." "I think the future is to push this in a high throughput screening platform to develop new drugs," he continued.

IRSF recently provided funding to support a new program for Rett syndrome-specific drug development, titled the "Selected Molecular Agents for Rett Therapeutics" (SMART) Initiative. The SMART Initiative will assemble a collection of brain-specific drugs that target select biological mechanisms important in RTT and make the compound collection available to researchers worldwide. IRSF's Chief Scientific Officer, Dr. Antony Horton said "IRSF has been proactive in moving iPSC technology forward for the purpose of drug screening, which is aligned well with the new SMART Initiative." "With the publication of this study, a major hurdle has been overcome in our quest to develop and test new medicines for the benefit of people living with Rett syndrome." added Dr. Horton.


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Contact: Stephen Bajardi
917-267-4503
International Rett Syndrome Foundation
Source:Eurekalert

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