Research Objective This project investigates how normal tau protein protects aging neurons. We'll use aging human neurons to understand how tau helps repairing DNA and why this protection fails disease.…
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 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 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 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…
Research Objective allogeneic HSC-engineered iNKT (HSC-iNKT) cells Impact treatment for COVID-19 Major Proposed Activities Milestone 1. Production of AlloHSC-iNKT and UHSC-iNKT cellsMilestone 2. Characterization of the AlloHSC-iNKT and UHSC-iNKT cellsMilestone…
Research Objective We expect to generate iPSC derived NK cells expressing a CAR against SARS-cov2 that could be used as an off-the-shelf therapy for COVID-19 Impact The proposed studies will…
Research Objective To develop and characterize meACE-2-CAR-IL15 NK cells expressing a mutated ACE2 and IL-15, allowing specific killing of SARS-CoV-2-infected cells and long in vivo persistence of the engineered cells.…
