Team Leader: Benjamin Prosser, PhDASO Leadership Drug Repurposing Leadership Leadership ASO CRISPR Drug Repurposing Gene Replacement ENDD has partnered with Panorama Medicine to repurpose FDA-approved compounds for both STXBP1 and SYNGAP1 disorders. After screening thousands of compounds, we have identified several FDA-approved lead candidates that are blood-brain barrier penetrant and can upregulate target gene expression. [...]
Team Leader: Giulia Pavani, PhDHuman Stem Cell Model Development Leadership Leadership Human Stem Cell Model Development The ability to generate human induced pluripotent stem cells (hiPSCs), which are biochemically and morphologically like embryonic stem cells, is a paradigm shift in human disease modeling. Now researchers can generate hiPSCs from non-disease persons as well as from [...]
Team Leader: Elizabeth Heller, PhDCRISPR Leadership Leadership CRISPR Dr Elizabeth Heller, whose niece has SYNGAP1 disorder, leads the CRISPR-related therapeutic development at ENDD. Her laboratory studies the epigenetic mechanisms that regulate gene expression as an underlying etiological factor for neuropsychiatric disorders, such as addiction and depression, as well as neurodevelopmental disorders. At ENDD, her group [...]
Team Leader: Mika HouserovaGene Replacement Leadership Our team Davidson Lab Gene Replacement Beverly Davidson, PhDGene Replacement Leadership Leadership Biomarker Gene Replacement Many neurodevelopmental disorders are the result of loss of one functioning copy of a critical gene – a condition referred to as haploinsufficiency. One way to treat haploinsufficiency is to add back a functional [...]
