Stage of Program: Basic or Applied Basic Research (DISC1 and many 1.0 Projects)
Enhanced Branching Morphogenesis and Pluripotent Cell Lineage Differentiation for Pediatric Regenerative Therapies
Research Objective Approximately 20,000 babies are born annually with kidney disease; the long-term outcome is poor. These studies address new ways to develop mini-kidney structures for transplantation to induce repair. Impact ~85% of people on the organ waitlist are in need of a kidney and there are insufficient donors. There is a pressing need to […]
Targeting cancer stem cells with nanoparticle RNAi delivery to prevent recurrence and metastasis of ovarian cancer
Research Objective Our objective is to develop novel treatments for women with ovarian cancer, specifically treatments that target stem cells to reverse drug resistance. These will treat recurrent metastatic disease. Impact We aim to use nanoparticles to make ovarian cancer stem cells more drug sensitive, less invasive, and less likely to regrow tumors and metastasize. […]
Human Pancreatic Cancer Stem Cells: Developing a Novel Drug for Cancer Eradication
Research Objective We will use human pancreatic cancer (PC) stem cells to show 1 inhibits proliferation, self-renewal and cell viability. This paradigm is transformational for anti-cancer drug discovery for patients. Impact Pancreatic cancer (PC) kills >40,000/yr in the US. PC is a major unmet medical need. Use of PC stem cells in development of 1 […]
Optimizing self-renewal signaling kinetics to stabilize ex vivo hematopoietic stem cell expansion
Research Objective We aim to develop conditions for stable expansion of blood stem cells outside of the body Impact Blood stem cells are a rare but necessary cell type for curative bone marrow transplantation and related gene therapies. Stable blood stem cell expansion will increase therapy availability and success Major Proposed Activities Validate a fully […]
Identification of antigenic neo-epitopes from in vitro reprogrammed human tissue precursors for regenerative therapy
Research Objective This study examine potential immunologic changes caused by cellular reprogramming that could present a barrier to clinical application of regenerative therapies. Impact Identification and evaluation of immunologic changes caused by cellular reprogramming provides critical information to maximize the efficacy and safety of regenerative cellualar therapies. Major Proposed Activities Identify changes to the repertoire […]
Development of treatments to improve healing of ischemic wounds
Research Objective We aim to develop an angiogenic proteoglycan mimic that will protect tissue from rapid degradation, and in conjunction with EPCs promote angiogenesis in order to accelerate ischemic wound healing. Impact As a treatment, LXW7-DS-SILY combined with a collagen scaffold and EPCs will accelerate healing of ischemic diabetic foot ulcers and reduce limb amputation […]
Novel metabolic labeling method for tracking stem cells to irradiated salivary glands using PET
Research Objective This project aims to develop a sensitive and non-invasive method for tracking stem cells in clinical trial, without the need for genetically engineered reporters or long-lived radioisotopes. Impact The ability to see follow stem cells over time, as they engraft, will make it possible to predict response to stem cell therapy and understand […]
Generation of human airway stem cells by direct transcriptional reprogramming for disease modeling and regeneration
Research Objective We will generate human airway stem cells by direct transcriptional reprogramming of fibroblasts. We will use these induced airway stem cells to model motile cilia disease in a dish. Impact Generating airway stem cells through reprogramming will create a scalable and editable cell line from which we can derive airway epithelium, thus enabling […]
An exosome-based translational strategy to mitigate Alzheimer’s disease neuropathology
Research Objective These studies will determine whether stem cell derived exosomes (nano-scale vesicles) can be used to treat the symptoms of Alzheimer’s disease (AD). Impact Our stem cell-derived exosome therapy will provide a viable approach to ameliorate the relentless progression of AD that severely impacts quality of life for millions of patients and their families. […]
Reprogramming human stem cells for blood cell generation
Research Objective To create a universal donor blood cell line that can be used to produce human red blood cells for transplantation. Impact Successful completion of this work would create a safe, unrestricted source of universal donor human blood cells that could be used to improve healthcare and save lives throughout the world. Major Proposed […]