Human Stem Cell Use: iPS Cell
Hypoxia-specific Production of Exosomes from iPSC-derivatives for Myocardial Repair
Research Objective A lead therapeutic candidate will be selected: 1) exosomes from hypoxia-injured iPSC-derived cardiomyocytes (iCMs), 2) exosomal miRNA cluster, and 3) siRNA inhibition of exosomal target gene, Notch3. Impact Effective targeted therapy to restore the injured and vulnerable myocardium is urgently needed to reduce the high mortality of HF patients. Promising discovery of iPSC […]
Small Molecules to inhibit Nemo-like Kinase for Treatment of Diamond Blackfan Anemia
Research Objective We propose to study small molecules that inhibit Nemo-like Kinase, to improve the production of red blood cells in bone marrow stem cells of children with Diamond Blackfan Anemia (DBA). Impact If small molecule NLK inhibitors are identified that are effective in improving the anemia of DBA and nontoxic, then treatment and transfusions […]
iPSCs as a screening tool to predict risk of nonalcoholic fatty liver disease
Research Objective The objective of this proposal is to established undifferentiated iPSCs as a diagnostic tool for the prediction of nonalcoholic fatty liver disease onset and severity. Impact Despite the widespread estimated prevalence of NAFLD, there are currently no tools available to predict likelihood of NAFLD susceptibility beyond standard clinical and demographic information. Major Proposed […]
Building a hiPSC-based biopacemaker
Research Objective A proof-of-concept biopacemaker constructed by bioprinting hiPSC-derived pacemaking cells and support cells based on the blueprint of the native pacemaking tissue of a large mammalian heart. Impact A hiPSC-based biopacemaker bioprinted using a design of the native pacemaking tissue in the heart, with protective electrical and mechanical insulations, can better sustain the pacemaking […]
Combating COVID-19 using human PSC-derived NK cells
Research Objective We propose to generate NK cells with enhanced immunity from gene-edited human PSCs and use the resultant NK cells to kill SARS-CoV-2-infected cells to combat against COVID-19. Impact The use of gene-edited hPSCs as a source for genetically engineered NK cells will allow us to generate effective immunotherapy for COVID-19 that has no […]
Development of COVID-19 Antiviral Therapy Using Human iPSC-Derived Lung Organoids
Research Objective To develop a new therapy for COVID-19 using human iPSC-derived lung organoids that targets SARS-CoV-2 protease known as the virus’ “Achilles Heel” Impact Our work, if successful, will bring a class of new drugs directly targeting viral enzyme and open the door for future COVID therapies. Major Proposed Activities Complete synthesis and testing […]
Human-induced pluripotent stem cell-derived glial enriched progenitors to treat white matter stroke and vascular dementia.
Research Objective This grant proposes development of a stem cell based therapy that is derived from human induced pluripotent stem cells. These cells are in the form of a brain support cell, an astrocyte. Impact The cell candidate will treat vascular dementia, the second leading cause of dementia, and stroke by overcoming a bottleneck in […]
Optimization of a human interneuron cell therapy for traumatic brain injury
Research Objective A cell therapy product comprised of inhibitory neurons that can migrate, integrate and restore neurologic function after traumatic brain injury. Impact Traumatic brain injury Major Proposed Activities Examine the most effective dose and safety profile of human iPSC-derived MGE cells grafted into rodent hippocampus. Determine whether human iPSC-derived MGE cells mature into appropriate […]
Development of a SYF2 antisense oligonucleotide (ASO) treatment for ALS
Research Objective We will develop an antisense oligonucleotide, or DNA therapy for diverse forms of amyotrophic lateral sclerosis (ALS). Impact ALS is fatal and incurable, and if successful, we will develop a treatment that slows or stops ALS progression across a broad range of patients. Major Proposed Activities Selection of the lead drug by testing […]
Human iPSC-derived chimeric antigen receptor-expressing macrophages for cancer treatment
Research Objective These studies will produce a new CAR-targeted iPSC-derived macrophage-based cell therapy product for treatment of refractory malignancies such as ovarian cancer. Impact These studies eliminate a bottleneck in macrophage production and enable these cells to be engineered and manufactured in a standardized, off-the-shelf manner, rather than on a patient-specific basis. Major Proposed Activities […]