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 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 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 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 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.…
Therapeutic Candidate or Device Autologous CD34+ cells transduced ex vivo with the BCH_BB694 LCR(-HS4) bGp D12shmiR lentiviral vector Indication Sickle cell disease with severe phenotype. Therapeutic Mechanism Silencing of beta-sickle…
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