The International Rett Syndrome Foundation (IRSF) announced today that it is awarding over $650,000 to support eight cutting-edge projects that aim to accelerate translational research to develop treatments for Rett syndrome. IRSF is the world's largest private source of funding for biomedical and clinical Rett syndrome research. Since 1998, IRSF has cumulatively funded over $24M in high quality, peer-reviewed basic and translational research grants that have significantly advanced Rett syndrome research towards finding a cure.
IRSF's Translational Research grant program includes two types of awards, which are the Help Accelerate Rett Therapeutics (HeART) and the Advanced Neurotherapeutic Grant of Excellence (ANGEL) awards. The funding from these grant awards will provide for early and late stage translational research to treat and reverse Rett syndrome (RTT).
The awarded projects from the first round of applications to the Translational Research grant program are geared towards drug discovery and development efforts as well as preparing for later stages of translational research. These funded projects fall into four main categories: (1) the development and testing of potential therapeutic compounds in animal models of Rett syndrome, (2) the development of outcome measures in humans that will be used in future clinical studies, (3) the development of a method for treatment, and (4) the development of a stem cell-based experimental system to be used in high-throughput drug screens. Together, they are in alignment with the objectives of IRSF's Translational Research grant program.
Stephen Bajardi, the executive director of IRSF, commented, "IRSF is pleased with the quality of these research projects and the significant steps they represent in moving Rett syndrome translational research forward." John M. Bissonnette, MD, Oregon Health Sciences University
Serotonin and small molecule treatment of respiratory disorders in a mouse model of Rett syndrome
New Translational Research Awards
Qiang Chang, PhD, of University of Wisconsin-Madison
Establishing Neurons Differentiated from an Isogenic Pair of Rett Syndrome iPSC lines as Cell-Based Assay for Future Drug Screens
Jenny Downs, PhD, Curtin University
Daily physical activity in girls and women with Rett syndrome: An important outcome for clinical trials
Steven J. Gray, PhD, University of North Carolina at Chapel Hill
Development of Optimized AAV Vectors for Intra-CSF Administration in Rett mice
Walter Kaufmann, MD, Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
Development of a Behavioral Outcome Measure for Rett Syndrome
Lucas Pozzo-Miller, PhD, University of Alabama-Birmingham
IGF-1 and TrkB Agonists as BDNF Mimetics for the Reversal of Dendritic Spine Pathologies and Network Hyperexcitability in the Hippocampus of MeCP2 Mutant Mice
Jay R. Shapiro, MD, Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
Treatment of Osteoporosis in Murine Rett Syndrome Models: A Comparison of Zoledronic Acid vs. Teriparatide on Osteoblast Function, Gene Expression and Bone Mass
Huda Y. Zoghbi, MD, Baylor College of Medicine
Therapeutic intervention to modulate the GABAergic and cholinergic systems in animal models of Rett syndrome
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