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 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…
Therapeutic Candidate or Device Human placental hematopoietic stem cell derived natural killer cells (CYNK-001) Indication SARS-CoV-2 positive patients requiring hospital admission and have any 2 out of 3 symptoms: fever,…
Therapeutic Candidate or Device Novel testing of a cell based therapy (Mesenchymal Stromal Cells) for respiratory failure from ARDS. Indication COVID-19 positive or negative ARDS patients Therapeutic Mechanism It will…
