Novel methods to eliminate cancer stem cells
Research Objective Our goal is to develop and optimize novel drugs that can attack blood cancer stem cells. These drugs interfere with a target protein, and will prevent relapse of…
Therapeutics to overcome the differentiation roadblock in Myelodysplastic Syndrome (MDS)
Research Objective This proposal will deliver a small molecule therapeutic candidate for the treatment of Myelodysplastic Syndromes and will act by inducing differentiation on mutated hematopoietic stem cells. Impact This…
Improving the efficacy and tolerability of clinically validated remyelination-inducing molecules using developable combinations of approved drugs
Research Objective The candidate is a fixed dose binary small molecule drug combination, consisting of two agents that act synergistically on a multipotent stem cell population in the CNS to…
Development of a new therapeutic for directing target specific stem cell migration and treatment
Research Objective A drug-stem cell combination therapy wherein the drug will direct and promote the delivery and distribution of stem cells to the disease site for the optimal therapeutic effect…
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…
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…
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…
Drug Development for Autism Spectrum Disorder Using Human Patient iPSCs
Research Objective We will use human patient induced pluripotent stem cell (hiPSC)-based models to screen for a drug that activates a transcription factor critical to the treatment of Autism Spectrum…
Small Molecule Proteostasis Regulators to Treat Photoreceptor Diseases
Research Objective We will discover small molecule compounds that correct disease in eyecups (retinal organoids) differentiated from patient iPSCs with photoreceptor diseases. Impact Our small molecule agents will provide new…
Stimulating endogenous muscle stem cells to counter muscle atrophy
Research Objective Intramuscular delivery of two repurposed FDA approved drugs will activate resident muscle stem cells. This therapeutic strategy will augment regeneration and restore strength to atrophied muscles. Impact Currently…
