Rettsyndrome.org announces new 2014 grants, investing over $3.1 million to accelerate research
December 23, 2014(Cincinnati, OH) The International Rett Syndrome Foundation - now doing business as Rettsyndrome.org - announces today that twelve (12) new grants were added to their research portfolio for a $1.2M investment, bringing the total 2014 research spend over $3.1M. Rettsyndrome.org is the leading private funder of Rett research, with more than $35 million invested to date. Rettsyndrome.org's strategy is to accelerate research toward new treatments for Rett syndrome by investing in basic, translational, and clinical research grants, which are vetted through a rigorous process of peer review by members of Rettsyndrome.org's Scientific Review Board (SRB). Those applications that have the highest scientific and programmatic merit are recommended to the Board of Directors for final funding decisions.
Two of the grants are funded by the Translational Research Program which funds early and late stage translational research to treat and reverse Rett syndrome (RTT). Rettsyndrome.org welcomes five new fellows to the Mentored Training Fellowship Program, which funds talented young post-doctoral researchers and aims to help them establish careers in Rett research.
Lastly, five grants from the Basic Research Program will fund investigators working to make new discoveries that will lead to a better understanding of the pathology of Rett syndrome and new avenues to treat the disorder.
With these new 2014 projects, Rettsyndrome.org continues to cover the spectrum of Rett syndrome research, in hopes of accelerating research from basic discovery, where ideas are made, to translating them into medicines for future clinical testing and treatments. These research grants are not restricted by geographic boundaries, and two new awards were given to researchers from Italy and the United Kingdom.
Rettsyndrome.org also announces that they will partner with the International Foundation for CDKL5 Research (IFCR) once again, to cost-share one of the newly awarded fellowships. With this continuing collaboration, both organizations can "share in funding this common endeavor that may have important implications for both CDKL5 and MECP2." said Katheryn Elibri Frame, DO, President and Co-founder of IFCR.
Rettsyndrome.org's Chief Science Officer, Steve Kaminsky, PhD, comments on the research strategy: "This year we had a great response to our Call for Proposals, and through the peer review process and programmatic planning, we have identified 12 research projects that will develop new ideas we hope to be translated to medicines. With the completion of two clinical trials and several others still underway, these are truly exciting times for the Rett community. These 12 new research projects add to our research investments, and we are hopeful that they build upon our portfolio and research strategy. We appreciate all of your support and look forward to this research adding to the growing knowledge base that will aid in correcting Rett biology."
New Fall 2014 Rettsyndrome.org Awards
HeART
- Alexander Kabanov, PhD, University of North Carolina at Chapel Hill
"Brain Delivery of BDNF via novel Nano-formulation for Treatment of Rett Syndrome" - Frank Menniti, PhD, Mnemosyne Pharmaceuticals, Inc.
"Effects of NMDA receptor modulators on network activity in a human iPSC-derived model of cortical dysfunction in Rett Syndrome"
- Lucas Pozzo-Miller, PhD, The University of Alabama at Birmingham
"Analogs of (1-3)IGF-1 (glypromate) for the improvement of hippocampal dysfunction in femaleMecp2 heterozygous mice: a preclinical trial for Rett syndrome"
- Breanne Byiers, PhD, University of Minnesota
"Assessing oculomotor function in Rett syndrome using integrated EEG and eye tracking technology" - Claudia Fuchs, PhD, University of Bologna, Italy
"Drug Therapy Targeted to Core Molecules in Neural Plasticity Cascades: A Promising Tool for the CDKL5 Variant of Rett Syndrome" - Janine Lamonica, PhD, University of Pennsylvania
"Targeting Protein Degradation Pathways to Treat RTT" - William Renthal, MD, PhD, Harvard Medical School
"Characterization of a Novel Activity-Dependent Phosphorylation Site on MECP2" - Xin Xu, PhD, The University of Alabama at Birmingham
"Molecular Mechanisms of Homeostatic Synaptic Plasticity in Mecp2 KO Neurons"
- Michael Brenowitz, PhD, Albert Einstein College of Medicine
"A biophysical basis for cellular and developmental regulation by MeCP2" - Darren Goffin, PhD, University of York
"Sensory Neural Network Alterations in Rett Syndrome" - Peng Jin, PhD, Emory University
"Consequences of altered DNA Methylation/hydroxymethylation caused by the loss of MeCP2 in neurons" - Jonathan Kipnis, PhD, University of Virginia
"Immune pathology and bone marrow transplantation in disorders of MeCP2 overexpression" - Lucas Pozzo-Miller, PhD, The University of Alabama at Birmingham
"Inhibitory interneuron dysfunction in network activity in female Mecp2 mice"
- Daniela Brunner, PhD, PyschoGenics, Inc
"Scout Program: A Drug Discovery Screen in a Mouse Model of Rett Syndrome" - Walter Kaufmann, MD, Boston Children's Hospital
"A Phase 2b placebo-controlled cross-over study of rh-IGF1 (mecasermin [DNA] injection) for treatment of Rett syndrome and development of Rett-specific novel biomarkers of cortical and autonomic function"
-end-
About Rettsyndrome.org Rettsyndrome.org is accelerating research for treatments and a cure for Rett syndrome. As the world's leading private funder of Rett research, we have funded more than $35 million in peer-reviewed research grants and programs to date. We are a 501(c)3 organization, earning Charity Navigator's most prestigious 4-star rating. We empower families to make a difference. Visit http://www.rettsyndrome.org to learn more, or call (800) 818-7388 (RETT).About Rett syndrome
Rett syndrome is a rare non-inherited genetic postnatal neurological disorder that occurs almost exclusively in girls and leads to lifelong impairments, affecting nearly every aspect of child's life: their ability to speak, walk, eat is effected, seizures develop and scoliosis occurs, and many develop irregular breathing patterns. Those diagnosed with Rett syndrome require maximum assistance with basic daily activities. The hallmark sign of Rett syndrome is near constant repetitive hand movements. Cognitive assessment in children with Rett syndrome is complicated, but they understand far more than they can communicate to us, evidenced by their bright and attentive eyes, and their ability to express a wide spectrum of moods and emotions.
International Rett Syndrome Foundation
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Two labs investigated whether the disruption of one protein's condensate-forming ability contributes to Rett syndrome.
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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.
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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
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