Late-stage development of AS-241, an UNC13A Targeting Antisense Oligonucleotide treatment for Amyotrophic Lateral Sclerosis, for IND-enabling studies

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• Post published:May 25, 2026
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Microglia replacement therapy for CSF1R-related Leukoencephalopathy

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• Post published:May 25, 2026
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CRISPR/Cas9-mediated gene editing of Hematopoietic stem and progenitor cells for Friedreich’s ataxia

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• Post published:May 25, 2026
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Development of an AAV Epigenetic Gene Therapy for Intractable Chronic Pain Disorders

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• Post published:May 25, 2026
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Dissecting cell-specific genetic and molecular drivers of amyotrophic lateral sclerosis for therapeutic insights

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• Post published:May 25, 2026
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Transplantation of human forebrain assembloids as a platform for therapeutic screening in neurodevelopmental disorders

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• Post published:May 25, 2026
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Advancing Therapeutic Discovery Through Multimodal Analyses of Genetic and Cellular Mechanisms in Neuropsychiatric Disorders

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Reversing age-dependent neurodegeneration by elimination of RNA pollution

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Tracing the metabolic basis of neurological disorders

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Modeling the genetic basis of psychopathology in schizophrenia and autism

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Research Objective We propose to apply scalable platforms for the characterization of CNVs and genes coupled with detailed characterizations of neurodevelopment of the 4 major CNV loci in model systems…

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