Project Objective: Proof of Concept

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 […]

Stem cell-derived extracellular vesicles to reverse radiation-induced brain injury

Research Objective These preclinical studies will discover the efficacy of stem cell-derived, nanoscale, extracellular vesicles (candidate) to treat adverse effects of cancer therapy on brain function and cognition. Impact Stem cell-derived extracellular vesicles will address the confounders of stem cells (tumors, immunorejection, immunosuppression) & mitigate debilitating side-effects of cancer therapy on the brain. Major Proposed […]

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 […]

Targeting Critical Regulators of Cancer Stem Cells

Research Objective We will develop a small molecule inhibitor that blocks the growth of human pancreatic cancer and AML cancer stem cells in vitro and in vivo. Impact This work will lead to a new treatment for cancer stem cell driven diseases such as AML and pancreatic cancer. In addition, it will improve the prognosis […]

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 […]

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 […]

Transplantation of Pluripotent Stem Cell Derived Microglia for the Treatment of Adult-onset Leukoencephalopathy (HDLS/ALSP)

Research Objective We propose to investigate the transplantation of pluripotent stem cell derived microglia as a potential therapy for the devastating neurological disease; Adult-onset leukoencephalopathy (ALSP/HDLS). Impact The most immediately impacted condition will be ALSP. However, further examination of the safety of human microglial transplantation will have broad implications for many neurodegenerative disorders Major Proposed […]