Research Objective We seek to develop a more accessible method of genome editing of blood stem cells directly in the body, so that the treatment can be safer and less…
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 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.…
Research Objective We will build a foundational model and experimental platform to catalog all genes that promote and protect against PAH, with the potential to extend to many other developmental…
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…
Research Objective Our work will yield an improved stem-cell based embryo model that we will explore with various omics approaches and genetic screens to gain insights into the pathways that…
Research Objective This research will allow the targeting of airway stem cells for long lived gene therapy for Cystic Fibrosis and for other airway diseases Impact We will overcome the…
Research Objective The objective of this proposal is to develop a human retinal organoid model of adRP to gain insights in pathogenesis and assess clinically relevant approaches to restore RHO…
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 We focus on hiPSCs pluripotency and neurodifferentiation to develop a novel framework to allow simultaneous identification of multiple interactions between proteins and between proteins and the genome Impact…
