Stage of Program: Basic or Applied Basic Research (DISC1 and many 1.0 Projects)
Engineering AAV capsids for transduction of neural and muscle stem cells
Research Objective The studies will identify and characterize new gene therapy vectors able to deliver gene editing components to stem cells. to enable treatment of diseases involving both muscle and brain. Impact New AAV capsids that target muscle and neural stem cells will enhance the number of neurological diseases able to be treated with AAV-based […]
Drivers of trophoblast stem/progenitor cell identity in human placenta
Research Objective This proposal will elucidate the mechanisms that identify a stem/progenitor cell population in the human placenta. Impact This proposal addresses our current knowledge gap on human placental development related to formation of the maternal/fetal interface during pregnancy, and placenta-associated pregnancy complications. Major Proposed Activities Identification of DNA regions, called enhancers, regulating placenta stem/progenitor […]
Generation of cortical organoids with tunable areal identities by spatial engineering of morphogens
Research Objective This project is to identify molecular mechanisms for generating different cortical regions using a novel hydrogel platform and brain organoids. Impact The hydrogel technology to establish localized signals and resulting enhanced cortical organoids would advance 3D human stem cell cultures as tools for biomedical innovation. Major Proposed Activities Hydrogel optimization for signaling molecule […]
Development of Anti-COVID RNAi Therapeutics Using Human iPSC-Derived Alveolar Epithelial Cells
Research Objective To optimize a new approach to deliver Anti-COVID siRNAs into human iPSC-derived lung cells that can selectively kill the COVID virus Impact Our proposal, if successful, will solve the siRNA delivery problem and rapidly open the door to Anti-COVID siRNA therapeutics. Major Proposed Activities Complete synthesis of a new delivery device called a […]
Using metabolic pausing for maintaining stable and high-quality pluripotent stem cells
An IPSC cell based model of macular degeneration for drug discovery.
Research Objective Towards a cure for age related blindness, we propose to create a human stem cell based model to screen for drugs that are protective against Age related macular degeneration (AMD). Impact With up to 11 million people in the United States affected by AMD, effective treatments against macular degeneration could address vision loss […]
Gingival mesenchymal stem cells as a novel treatment modality for periodontal tissue regeneration
Research Objective To develop a novel regenerative and adhesive hydrogel encapsulating patient's' gingival stem cell which can potentially be used as an adhesive dental hydrogel for periodontal tissue regeneration. Impact Upon successful completion, this project will introduce a promising treatment approach for maxillofacial defects presenting an innovative treatment modality for periodontal tissue regeneration. Major Proposed […]
A new phenotypic screening platform that identifies biologically-relevant targets and lead compounds for the treatment of Parkinson’s disease
Research Objective Demonstrate that our HitFinder™ library can be screened for phenotypic changes in A53T-IPSC-derived dopaminergic neurons and use a secondary handle to identify the targets responsible. Impact This technology combines phenotypic screening and target-ID eliminating the need to bias assays and/or screening libraries permitting application directly in iPSC-derived cells. Major Proposed Activities Prepare screening […]
IVD rejuvenation using iPSC-derived notochordal cells
Research Objective To identify a new therapeutic agent for disc regeneration using novel pluripotent stem cells and injectable beads that support differentiation and provide biomechanical strength. Impact If this study is successful, we will be able to bring completely new biologically and biomechanically relevant solution to degenerated intervertebral discs. Major Proposed Activities To optimize stem […]
iPSC-Derived Smooth Muscle Progenitors for Treatment of Abdominal Aortic Aneurysm
Research Objective To assess the therapeutic effect of human induced pluripotent stem cell (iPSC)-derived smooth muscle progenitors (pSMCs) for treatment of abominal aortic aneurysm (AAA). Impact Currently, there are no pharmacologic therapies for AAA. If successful, delivery of autologous pSMCs to the site of AAA will halt or reverse the progression towards a rupture-prone aneurysm. […]