Where Your Donations Go
We can only do this with your support and generous donations.
Our challenge is to find answers. Without our funding, the research will end here.
We have made agreements with 2 world renowned children’s hospitals to find answers fast!
Both Texas Children’s Hospital and Nationwide Children’s hospitals are going through great efforts to find answers and a cure.
All research teams that are funded by the iDREAM For a Cure Foundation, are required to collaborate with International teams we have partnered with in order to eliminate repeated studies and to quicken our goal to find a cure for all IRF2BPL Related Disorders. Our research institutions have protocols in place to share patient cell samples, reagents, characterized and generated IRF2BPL mice and all data from IRF2BPL studies.
Texas Children’s Hospital
Understanding the role of IRF2BPL in neurological disease.
The goal of our funding at this lab is to unravel the biological function of IRF2BPL that may lead to identification of targets for therapeutic intervention. Via fly models, Dr. Marcogliese has discovered a primary pathway for IRF2BPL pathogenesis. Based on this pathway, the Bellen lab is currently testing several drugs for repurposing, for use in our patient population.
In addition to characterizing and generating IRF2BPL mutant drosphila flies for testing, this lab has also successfully characterized Irf2bpl mutant mice and have generated Irf2bpl knockout mice to further study IRF2BPL. Since 2020, they have focused on a pilot drug screen to determine if drugs that impact their found mechanism of action, will have positive effects on the mice population they have derived.
Nationwide Children's Hospital
Assessing Gene Therapy and other therapeutic approaches for IRF2BPL mutation caused disease in vitro and in vivo.
Dr. Meyers team at Nationwide Children’s Hospital has, under our funding, designed multiple gene therapy vectors to restore IRF2BPL expression to physiological levels as well as created small-hairpin RNA vectors that will allow reducing the expression levels of IRF2BPL. Both vector types will be tested in wild type mice for safety and to verify whether reduced expression of wild type IRF2BPL causes a disease phenotype similar to what is found in human patients. They are using co-culture assays to test novel therapeutics and gene therapy efficacy.
Most encouraging, this lab is also studying the effects of drugs currently in clinal trials for other neurodegenerative diseases, as well as already FDA approved drugs on our patient population. Dr. Meyer’s lab has recently confirmed that a compound they are testing seems to have a promising effect on several patient astrocytes. This compound is already in clinical trials for other diseases, which is good and could mean should it remain promising, that it could be a relatively quick way forward towards clinical trials.
Meet Our Researchers
Dr. Hugo J Bellen, D.V.M., Ph.D.
Distinguished Service Professor
Baylor College of Medicine- Neurological Research Institute: Departments of Molecular and Human Genetics and Neuroscience
Dr. Paul C Marcogliese, Ph.D.
CIHR Postdoctoral Fellow
Baylor College of Medicine- Neurological Research Institute: Department of Molecular and Human Genetics
Dr. Kathrin Meyer,
Assistant Professor & Principal Investigator
Center for Gene Therapy, The Research Institute at Nationwide Children’s Hospital; College of Medicine, Department of Pediatrics, The Ohio State University
Dr. Shrestha Sinha Ray, PhD
Center for Gene Therapy, The Research Institute at Nationwide Children’s Hospital
Legal Charity Status
The iDream For A Cure Foundation is a project of The Giving Back Fund, a 501©(3) non-profit organization with federal tax-exempt status as a public charity, ID 04-3367888.
iDream For A Cure
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