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…
Research Objective We develop a powerful platform to correct gene expression defects caused by haploinsufficiency and will show its ability to identify novel targets using iPSC-derived neurons and cortical organoids.…
Research Objective This proposal leverages pluripotent stem cell models to understand the principles by which changes in gene and protein dosage affect human neural and facial progenitor development and cause…
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…
Research Objective The proposed aims will enable the generation, purification and characterization of enteric neurons from diverse hiPSCs and assessment of their efficacy for cell therapy in GI motility disorders.…
Research Objective Here we will study the role of ataxin-3 alternative polyadenylation in the pathogenesis of ALS, and test if ASOs can reduce distal polyadenylation of ataxin-3 to rescue ALS…
Research Objective Our goal is to develop new gene editing methods for producing universal, off-the-shelf, therapeutic T cells from induced pluripotent stem cells (iPSC) that can be applied to a…
Research Objective Development-inspired differentiation will enable efficient and specific generation of tenocytes that can repair tendon injury, restore dysfunctional tissue, and prevent long term effects. Impact This study will eliminate…
Research Objective Greenstone Biosciences will use patient-derived stem cells to discover safe and effective drugs for cardiovascular disease. Impact The use of patient-derived stem cells to discover novel drug targets…
Research Objective This proposal aims to develop a novel cell based vaccine for the treatment of cancer. The vaccine would be derived from stem cells and be personalized to the…
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