Research Objective We will test a novel target to augment endogenous muscle stem cell function and restore strength systemically in sarcopenia. Impact Sarcopenia can be highly disabling and has limited…
Research Objective To test of exosomes (a tiny vesicle produced by stem cells) can be used to treat brain injuries rather than having to use the entire cell. Impact Not…
Research Objective Validating the cardiac MPS as tool to predict cardiac toxicity, including modeling drug-drug interaction of polytherapy for the treatment of SARS-CoV-2. Impact Our cardiac MPS will identify polytherapy…
Research Objective We propose to evaluate both gene addition with viral vectors and CRISPR-based gene editing of a patient's own bone marrow stem cells as a curative therapy for autosomal…
Research Objective This DISC2 tests a strategy to target survival and self-renewal in glioma stem cells (GSC), and will identify a therapeutic candidate for safety, dosing and efficacy testing. Impact…
Research Objective This proposal will develop a cell-based therapy that can restore vision in retinal degeneration associated blindness such as Stargardt disease and age-related macular degeneration. Impact Chemically induced method…
Research Objective Development of a CRISPR genome editing therapy for ALS caused by C9orf72 and the delivery vehicle required to bring this novel therapy to patients. Impact A cure for…
Research Objective With a single administration to an individual, lentiviral vectors that selectively transduce B cells in vivo and express highly potent anti-HIV-1 proteins to suppress HIV-1 replication throughout life…
Research Objective The therapeutic candidate we aim to discover under this award is clomipramine, an antidepressant that might help reduce a harmful protein in the nerve cells of patients with…
Research Objective This personalized chimeric antigen receptor (CAR) T cell therapy will attack a wider range of cancer cells, and block cancer's defense mechanisms to empower the immune system to…
