“Of all the childhood genetic neurologic disorders of the brain, Angelman syndrome may be the single best candidate for developing a definitive treatment (aka a cure).”~Dr. Arthur Beaudet The Foundation for Angelman Syndrome Therapeutics (FAST) US and FAST AU are excited to announce joint funding to Dr. Beaudet to generate mouse lines expressing human UBE3A
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written by Allyson Berent, DVM DACVIM When we refer to gene therapy in Angelman Syndrome (AS), we are typically referring to a “viral delivery” or “virus vector” of the missing gene (UBE3A) or the missing protein (UBE3A protein) into the brain. AS is caused when the UBE3A gene is either missing or not functioning properly;
FAST (Foundation for Angelman Syndrome Therapeutics) has funded two research labs at the University of California, Davis (UC Davis) to further investigation into gene editing for Angelman Syndrome. One funded project continues the evaluation of Artificial Transcription Factors (ATFs or Zinc Fingers) for the treatment of Angelman Syndrome (AS) in Dr. David Segal’s laboratory (pictured
How FAST Is Changing The Landscape Of Angelman Research When you set out to cure an ‘incurable’ genetic disorder, you need three things: a comprehensive, well thought out plan; a dedicated and passionate community; and sufficient, often substantial funding. With a grant from the Marnier Lapostolle Foundation of $5.8 million, the Foundation for Angelman Syndrome
Ketone ester supplementation attenuates seizure activity, and improves behavior and hippocampal synaptic plasticity in an Angelman syndrome mouse model
Read more about the exciting work that has earned the Segal Lab $1.1 Million for research.
Additional Gene Therapy Approach For Angelman Syndrome Explored By Paula Evans The Foundation for Angelman Syndrome Therapeutics (FAST) is delighted to announce that research FAST has funded, through the support of our donors, has secured a $1.1 Million grant from the California Institute for Regenerative Medicine, to develop an additional gene therapy approach for the
RDr. Dindot’s 3 goals, which have been the focus of his AS research through the FIRE initiative, are;
Mechanisms regulating imprinting of UBE3A in neurons, Identification of new therapeutic potentials for AS using high
throughput assays and Generation of new animal models of AS.
Dr Anne Anderson of the FAST FIRE team works on evaluating seizure and behavioral phenotypes (characteristics) in AS mice and how this information can be used in preclinical studies.