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DSF Funded Research

DSF Funded Research

photos of researchers for Circuit-selective Whole SCN1A Gene Delivery for Dravet Syndrome

Circuit-selective Whole SCN1A Gene Delivery for Dravet Syndrome

Boaz P. Levi, PhD- Allen Institute for Brain ScienceBryan B. Gore, PhD- Allen Institute for Brain ScienceJohn K. Mich, PhD- Allen Institute for Brain ScienceTim Jarsky, PhD- Allen Institute for Brain Science  Circuit-selective Whole SCN1A Gene Delivery for Dravet Syndrome Transformational Science Grant- $500,000 over 3 yearsThis grant funded with support from Marlins for Mason  […]

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Liu Goldberg 1

Base editing for the treatment and prevention of Dravet syndrome

Ethan M. Goldberg, MD, PhD- The Children’s Hospital of PhiladelphiaDavid R. Liu, PhD- MIT and Harvard University Base editing for the treatment and prevention of Dravet syndromeResearch Grant- $250,000,  2 years Summary from the Investigators: Dravet syndrome is caused by genetic mutations in the gene SCN1A that encodes the voltage-gated sodium channel subunit known as

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Xu Zhang, PhD

Selective Activation of Hippocampal Parvalbumin Interneurons via Focused Ultrasound Neuromodulation for Seizure Suppression in Scn1a+/- Mice

Xu Zhang, PhD – Boston Children’s Hospital, Harvard Medical School Selective Activation of Hippocampal Parvalbumin Interneurons via Focused Ultrasound Neuromodulation for Seizure Suppression in Scn1a+/- Mice Postdoctoral Fellowship – $75,000, 1 year Recipient of the Elliot Meskis Award for an Exceptional Postdoctoral Fellow which provides a $2,500 grant supplement for professional development. Summary from the

Selective Activation of Hippocampal Parvalbumin Interneurons via Focused Ultrasound Neuromodulation for Seizure Suppression in Scn1a+/- Mice Read More »

Petra Bencurova, PhD

Multi-Target Strategy for Dravet Syndrome: Enhancing Sodium Channels and GABA Receptors via miRNA Inhibition

Petra Bencurova, PhD; Royal College of Surgeons in Ireland Multi-Target Strategy for Dravet Syndrome: Enhancing Sodium Channels and GABA Receptors via miRNA Inhibition Postdoctoral Fellowship- $75,000/ 1 year Supported by Budgetdog, LLC Grant Summary from the Investigator: Dravet Syndrome (DS) is a rare and severe form of epilepsy in children that causes a variety of issues

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Kremer Tolner Rubinstein Goldberg

Molecular characterization of the therapeutic effect of exogenous NaV1.1

Eric J. Kremer, PhD- Institut de Génétique Moléculaire de MontpellierMoran Rubinstein, PhD- Tel Aviv UniversityElse A. Tolner, PhD- Leiden University Medical CenterEthan M. Goldberg, MD, PhD- The Children’s Hospital of Philadelphia Molecular characterization of the therapeutic effect of exogenous NaV1.1 Transformational Science Grant- $500,000 over 3 years Funding for this grant was supported by Dance

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Baraban Isom Parent grant

Therapeutic benefit of MGE progenitor cell transplantation in a rabbit model of Dravet syndrome

Lori L. Isom, PhD – University of MichiganScott Baraban, PhD – University of California San FranciscoJack M. Parent, MD – University of MichiganTherapeutic benefit of MGE progenitor cell transplantation in a rabbit model of Dravet syndromeTransformational Science Grant – $500,000, 3 years Grant Summary: Dravet syndrome (DS) is a devastating developmental and epileptic encephalopathy that

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Theyel grant

Abnormal ectopic action potentials in PV-INs: A novel pathophysiological mechanism in Dravet syndrome

Brian Theyel, MD, PhD – Brown UniversityAbnormal ectopic action potentials in PV-INs: A novel pathophysiological mechanism in Dravet syndromeResearch Grant – $250,000, 2 years Grant Summary: Dravet syndrome, which is caused by a mutation in the SCN1A gene, is a debilitating illness that emerges during the first year of life. It causes seizures, setbacks during development, speech and language

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Sanatani Connolly grant

Extended monitoring for cardiac arrhythmias in Dravet syndrome

Mary Connolly, MB, BCh, FRCPC(C), FRCP(I), FRCP(Edin) – The University of British ColumbiaShubhayan Sanatani, MD, FRCPC – The Universty of British ColumbiaExtended monitoring for cardiac arrhythmias in Dravet syndromeClinical Research Grant – $250,000, 2 years Grant Summary: Dravet syndrome (DS) is a rare serious condition associated with early onset of seizures that typically do not

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2021 2023 research grant graphics

DS-TIME: a multi-assessment approach to uncover the underlying mechanisms of sleep disturbances in children with Dravet syndrome

Cristina Reschke, PhD – RCSI University of Medicine and Health SciencesDS-TIME: a multi-assessment approach to uncover the underlying mechanisms of sleep disturbances in children with Dravet syndromeClinical Research Grant – $250,000, 2 years Grant Summary: Dravet syndrome (DS) is a very difficult to treat epilepsy syndrome characterized by seizures that start in the first years

DS-TIME: a multi-assessment approach to uncover the underlying mechanisms of sleep disturbances in children with Dravet syndrome Read More »

Hill grant

Mechanistic investigation in a novel mouse model of early-onset SCN1A-related disorders

Sophie Hill, PhD – Children’s Hospital of PhiladelphiaMechanistic investigation in a novel mouse model of early-onset SCN1A-related disordersPostdoctoral Fellowship – $75,000, 1 year Grant Summary: Mutations in the SCN1A gene most commonly cause reduced functioning of the Nav1.1 protein and lead to Dravet syndrome, a debilitating epilepsy disorder. However, a new class of SCN1A mutations

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