Dravet Syndrome Foundation Funds Five New Research Grant Awards totaling $1.4M

Dravet Syndrome Foundation announced the recipients of their 2024 research grants at their 15th annual Research Roundtable on December 5th in Los Angeles.

CHERRY HILL, NJ, UNITED STATES, December 18, 2024 /EINPresswire.com/ -- Dravet Syndrome Foundation (DSF) announced the recipients of their 2024 research grants at the 15th annual Research Roundtable held on December 5th in Los Angeles, California. DSF is proud to fund five new research grants totaling $1.4 million this year. Three of these grants are made possible through the support of Marlins for Mason (Prather Family), Dance for Dravet (Brennan and Odlaug Families), and Budgetdog, LLC. With this funding, DSF has now contributed over $10.5 million to research since its inception in 2009.

DSF Executive Director Mary Anne Meskis said, “We are incredibly grateful for the strong support from our donors and supporters, whose contributions are making a real difference in advancing progress for Dravet syndrome. This support underscores the seriousness of the disease and the dedication of our community to improving outcomes for those affected by Dravet syndrome. We eagerly anticipate the developments that will emerge from these grants.”

A DSF Transformational Science Grant was funded with support from Marlins for Mason (The Prather Family) for $500,000 over three years to researchers at the Allen Institute for Brain Science. Boaz P. Levi, PhD, Bryan B. Gore, PhD, John K. Mich, PhD, and Tim Jarsky, PhD will collaborate on the project titled “Circuit-selective whole SCN1A gene delivery for Dravet syndrome.” The grant will allow them to test a new targeted genetic therapeutic strategy in a mouse model of Dravet syndrome and investigate the impacts on signaling and genetic networks in the brain.

A second Transformational Science Grant for $500,000 was funded with support from Dance for Dravet (The Brennan and Odlaug Families) to a project titled “Molecular characterization of the therapeutic effect of exogenous NaV1.1.” This collaborative effort brings together Eric J. Kremer, PhD from the Institut de Génétique Moléculaire de Montpellier; Moran Rubinstein, PhD from Tel Aviv University; Else A. Tolner, PhD from Leiden University Medical Center, and Ethan M. Goldberg, MD, PhD from The Children’s Hospital of Philadelphia to delve further into understanding the mechanisms by which their novel therapeutic approach to genetic therapy for Dravet syndrome is effective, including the impacts on brain signaling, gene expression, and required duration of therapy.

A Research Grant for $250,000 was awarded to the project “Base editing for the treatment and prevention of Dravet syndrome” from Ethan M. Goldberg, MD, PhD from The Children’s Hospital of Philadelphia and David R. Liu, PhD from MIT and Harvard University. They will use a cutting-edge base editing technique, based on CRISPR/Cas9 gene editing technology that allows for correction of a single base pair mutation in the DNA; testing it in a mouse model of Dravet syndrome and assessing the impact on seizures, mortality, and neuronal signaling.

A Postdoctoral Fellowship was awarded to Xu Zhang, PhD at Boston Children’s Hospital and Harvard Medical School in the amount of $75,000 for the project “Selective activation of hippocampal parvalbumin interneurons via focused ultrasound neuromodulation for seizure suppression in Scn1a+/- mice.” This project will explore the novel approach of using focused ultrasound stimulation as a noninvasive, targeted therapy to modulate brain activity and reduce seizures. Dr. Zhang also received the Elliot Meskis Award which provides a $2,500 supplement to be used for professional development to an outstanding DSF postdoctoral fellow.

A Postdoctoral Fellowship was awarded to Petra Bencurova, PhD at the Royal College of Surgeons in Ireland in the amount of $75,000 funded with support from Budgetdog, LLC for the project “Multi-target strategy for Dravet syndrome: Enhancing sodium channels and GABA receptors via miRNA inhibition.” The project will focus on small molecules called microRNAs that may be able to modify expression of SCN1A or other related genes to act as a novel treatment to address symptoms of Dravet syndrome.

Grants are awarded through a competitive application and review process. Said DSF Scientific Director, Dr. Veronica Hood, “The 2024 DSF grant awards are representative of the immense progress that is occurring in the field, with all five grants focused on novel, targeted therapeutic approaches that each hold the potential to address symptoms and even modify the course of Dravet syndrome.”

You can read abstracts for each of the 2024 grant awards, as well as see past projects at https://dravetfoundation.org/dsf-funded-research/.

DSF has funded 71 research projects, totaling over $10.5M since 2009, making them the largest non-governmental funder of Dravet syndrome research, worldwide. Of those projects, over 72% have resulted in scientific publications, with DSF contributing to over 75 scientific publications either through direct involvement and/or financial support. DSF-funded researchers have subsequently received over $34M in NIH-level funding for projects related to Dravet syndrome.

Mary Anne Meskis
Dravet Syndrome Foundation
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