Genetic and Epigenetic Regulation of XIST and X-chromosome silencing in hiPSCs: Overcoming Barriers in Stem Cell-Based Therapies for Women’s Health

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Research Objective We study how the inactive X chromosome aberrantly reactivates in female pluripotent stem cells and develop ways to prevent it, enabling accurate modeling of female biology for research…

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Allele Prospector: Leveraging human genetic variation to enable therapeutic genome editing in hundreds of disease genes

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Research Objective We are building a foundational platform for developing genome editing technologies that increase patient coverage by 20-40X and can be applied to over 700 genetic diseases in diverse…

Continue ReadingAllele Prospector: Leveraging human genetic variation to enable therapeutic genome editing in hundreds of disease genes

Modeling Rett syndrome neurological disorder with human pluripotent stem cells to develop in cellulo screening platforms.

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Research Objective Study the neurological disorder Rett syndrome using neurons derived from genetically engineered human pluripotent stem cells and develop platforms to screen drugs for this disease in live cells.…

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Modeling and understanding alveolar hypoplasia in Down syndrome using iPSCs-derived alveolar type II cells

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Research Objective Understanding alveolar progenitor cell defects in T21 and the genes/pathways associated with them will allow for developing therapeutic approaches for individuals with DS. Impact Although trisomy 21 affects…

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An interactive data resource for hypothesis testing in stem cell single-cell gene expression and validation of the results with brain organoids

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Research Objective We are building a "virtual molecular microscope" where anyone can quickly visualize a very recent, high-throughput molecular assay, single-cell RNA-seq and spatial gene expression studies Impact Currently, a…

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Development of a stem-cell based approach to interpret global effects of genetic variants contributing to neurodevelopmental disease risk

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Research Objective We are developing a strategy to characterize the disease-relevance of hundreds of mutations across diverse genetic backgrounds using stem cells Impact Understanding how mutations impact cellular function can…

Continue ReadingDevelopment of a stem-cell based approach to interpret global effects of genetic variants contributing to neurodevelopmental disease risk