Advancement of a myotropic, liver-detargeting therapy for LGMD2i/R9

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• Post published:April 30, 2026
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A first-in-class CRISPR-CasX gene editor silencing APOC3 transcription for the treatment of Severe Hypertriglyceridemia

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• Post published:April 30, 2026
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Microglia replacement therapy for CSF1R-related Leukoencephalopathy

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TY1 and Semaglutide to Treat Cardiometabolic HFpEF

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CRISPR/Cas9-mediated gene editing of Hematopoietic stem and progenitor cells for Friedreich’s ataxia

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Noncoding RNA drug TY2 for arrhythmogenic cardiomyopathy

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Development of an AAV Epigenetic Gene Therapy for Intractable Chronic Pain Disorders

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Stem Cell-Based Cartilage Tissue Regeneration

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Autologous iPSC-derived progenitor smooth muscle cells for treatment of urinary incontinence

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Translational epigenomics: dissecting cell type-specific function of neuropsychiatric risk genes in vivo

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Research Objective Our objective is to enable scalable genetic screening to study how different cell types and epigenetic networks are impacted by risk genes implicated in human psychiatric disorders. Impact…

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