IRSF awards over $1.5 million for basic and translational research for Rett syndrome

December 19, 2011

Cincinnati, (OH) - The International Rett Syndrome Foundation (IRSF) announced today that it is awarding over $1.5M to support 18 new grants designed to study a variety of diverse topics from basic science and disease pathology to developing treatments and outcome measures for Rett syndrome. IRSF is the world's largest private source of funding for biomedical and clinical Rett syndrome research with the cumulative total of research spending of over $26M since 1998. It is the mission of IRSF to invest in these high quality, peer-reviewed basic and translational research grants to advance research towards new treatments for Rett syndrome.

IRSF's Grants & Research department encompasses two research programs: Basic Research and Translational Research. Under the Basic Research Program, the grant mechanisms cover Regular Research Grants and Postdoctoral Fellowships, which are designed to assist independent investigators and post-doctoral researchers establish careers in fields relevant to Rett syndrome research and to obtain future funding from other agencies. The Translational Research Program funds early and late stage translational research to treat and reverse Rett syndrome (RTT) using two grant mechanisms, Help Accelerate Rett Therapeutics (HeART) and the Advanced Neurotherapeutic Grant of Excellence (ANGEL), respectively.

The awarded basic research projects explore a range of topics from basic biology in gene regulation to understanding pathways involved in neuronal cell communication. Together, these types of studies will allow for identification of novel therapeutic targets in Rett syndrome. The second round of translational grants are focused on repurposing FDA-approved drugs for Rett syndrome, development and testing of potential therapeutic compounds, and development of outcome measures in humans that will be used in future clinical studies. Collectively, this year's awards will contribute to the different pathways into the drug development pipeline for Rett syndrome and move forward IRSF's scientific agenda "Research to a Reality".

Stephen Bajardi, the executive director of IRSF, commented, "IRSF is gratified to see the growing interest and focus on Rett syndrome research, as well as the global nature of these projects with five out of the eighteen grants coming from countries other than the United States."

New Translational Research Awards
New Basic Research Awards-Regular Research Grants New Basic Research Awards-Postdoctoral Fellowships
-end-
About Rett Syndrome

Rett syndrome (RTT), a developmental neurological disorder, occurs almost exclusively in females. RTT results in severe movement and communication problems following apparently normal development for the first six to 18 months of life. Characteristic features of the disease include loss of speech and purposeful hand use, repetitive hand movements, abnormal walking, abnormal breathing, slowing in the rate of head growth and increased risk of seizures. Current treatment for girls with RTT includes physical and occupational therapy, speech therapy, and medication for seizures. There is no known cure for RTT. In 2007, researchers heralded a major breakthrough by reversing RTT symptoms in mouse models. RTT is considered a "Rosetta Stone" that is helping scientists understand multiple developmental neurological disorders, and shares genetic links with other conditions such as autism and schizophrenia.

About the International Rett Syndrome Foundation.


IRSF is the world's leading private funder of basic, translational and clinical Rett syndrome research, funding over $26M in high-quality, peer-reviewed research grants and programs to date. Annually, IRSF hosts the world's largest gathering of global Rett researchers and clinicians to establish research direction and priorities while exchanging ideas and the most recent information. IRSF is the most comprehensive non-profit organization dedicated to providing thorough and accurate information about Rett syndrome, offering informational and emotional family support, and stimulating research aimed at accelerating treatments and a cure for Rett syndrome and related disorders. IRSF has earned Charity Navigator's most prestigious 4 star rating. To learn more about IRSF and Rett syndrome, visit http://www.rettsyndrome.org or call IRSF at 1-800-818-RETT (7388).

International Rett Syndrome Foundation

Related Rett Syndrome Articles from Brightsurf:

Proteins -- and labs -- coming together to prevent Rett syndrome
Two labs investigated whether the disruption of one protein's condensate-forming ability contributes to Rett syndrome.

Genetic editing milestone in mouse model of Rett Syndrome
A genomic error that causes Rett Syndrome, a serious lifelong neurological disorder, can be corrected in the brains of mice by rewriting the genetic instructions carried by the RNA.

Yale researchers find potential treatment for Rett Syndrome
An experimental cancer drug can extend the life of mice with Rett Syndrome, a devastating genetic disorder that afflicts about one of every 10,000 to 15,000 girls within 6 to 18 months after birth, Yale researchers report June 10 in the journal Molecular Cell.

Research team investigates abnormal neuron activity in Rett syndrome
Research by Billy Lau, a postdoctoral researcher working with Assistant Professor Keerthi Krishnan at the University of Tennessee, Knoxville, examines the time during which an adult female mouse first learns to recognize and respond to the distress cries of young mouse pups as an opportunity for the brain to rewire and learn again.

A dietary supplement improves skills of an atypical Rett syndrome patient
Administration of the amino acid L-serine, a dietary supplement, contributes to the improvement of the communicative and motor skills of a patient with a mutation that alters glutamate receptors.

A new drug shows preclinical efficacy in Rett syndrome
A new article published in the Cell Reports describes how a new drug is able to reduce the symptoms and activate the dormant neurons characteristic of Rett Syndrome in preclinical models.

X chromosome reactivation could treat Rett syndrome, other X-linked disorders
A study from a team of Massachusetts General Hospital investigators points toward a potential strategy for treating X-linked disorders -- those caused by mutations in the X chromosome -- in females.

Discovery fuels hope for Rett syndrome treatment
Vanderbilt University researchers have relieved symptoms of Rett syndrome in a mouse model with a small molecule that works like the dimmer switch in an electrical circuit.

Drug improves brain performance in Rett syndrome mice
A brain penetrant drug -- a small-molecule mimetic of BDNF, or brain derived neurotrophic factor -- is able to improve brain performance in Rett syndrome mice -- specifically synaptic plasticity in the hippocampus and object location memory.

Small-molecule therapeutic boosts spatial memory and motor function in Rett syndrome mice
Rett syndrome is a neurological disorder affecting learning and development, caused by a mutation in the MECP2 gene triggering decreased levels of brain-derived neurotrophic factor (BDNF).

Read More: Rett Syndrome News and Rett Syndrome Current Events
Brightsurf.com is a participant in the Amazon Services LLC Associates Program, an affiliate advertising program designed to provide a means for sites to earn advertising fees by advertising and linking to Amazon.com.