Project Objective: Tool/Resources/Bottleneck
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 […]
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 […]
Organoid Modeling of Human Cortical Microcircuits
Research Objective The proposed studies will develop three-dimensional cell culture methods for creating human brain neural circuits for disease research and drug discovery. Impact The proposed research will develop a new research platform for studying how neurons in the human brain function, how neurological disease subverts this activity, and how we might find new therapies. […]
Genome editing for causation and reversion of MPN-associated mutations in human hematopoietic stem cells
Research Objective Use gene editing to create tools for the study of mechanisms by which patient-observed mutations lead to myeloproliferative neoplasms. Impact Editing reagents will yield new insight into how acquired MPN-associated mutations cause disease by overproduction of various cell types and pave the way for gene editing therapies to reverse MPNs. Major Proposed Activities […]
Microenvironment based optimization of retinal induction using CRISPR-CAS9 reporter pluripotent stem cells as an expandable source of retinal progenitors and photoreceptors.
Research Objective To increase the efficiency of generating pure retinal progenitor cultures for use in transplantation and to probe general aspects of retinal development. Impact Our methods could increase the efficiency of obtaining transplantable patient specific induced pluripotent stem cell derived retinal cells for the treatment of blindness through cell replacement. Major Proposed Activities Make […]
Purification of Human Hematopoietic Stem Cells (HSCs) for Clinical Stem Cell Transplantation
Translational Candidate Two new monoclonal antibodies anti-CD34 and anti-CD90, and protocols to purify cancer-free and/or T cell free human HSC for clinical transplantation. Area of Impact The development of hematopoietic stem cell-based therapies, starting with rescue of metastatic breast cancer patients with their own cancer-free HSC Mechanism of Action The mechanism is using highly purified […]
Development of a low-cost, clinical-grade iPS maintenance medium for enabling stem cell therapy manufacturing
Translational Candidate HiDef-B8, an iPS cell maintenance medium designed to reduce stem cell GMP manufacturing costs and risk. Area of Impact HiDef-B8 addresses scale-up manufacturing, by being lower-cost ($300/L) and requiring fewer passages per week (1-2 as opposed to 3-5). Mechanism of Action HiDef-B8 underwent extensive empirical optimization and alternative component screening, focused on reducing […]
Generation of human universal donor iPS cells
Translational Candidate Universal donor cell that is a genetically-engineered iPSC clone and is equipped with a safety switch. Area of Impact Regenerative medicine including replacement therapies affected by immune rejection by host immune cells. Mechanism of Action The universal donor cell mitigates immune rejection by host immune cells and is equipped with a suicide gene […]
T-Pure: Peripheral Blood Processing Tool for Point of Care CAR-T Manufacturing
Translational Candidate Tool kit added directly to peripheral blood allowing for purification of T cell enriched product suitable for (CAR)-T cell generation. Area of Impact The goal is to generate a tool that can address one of the most expensive and rate-limiting steps in the production of genetically engineered cells Mechanism of Action Our application […]
Cell Villages and Clinical Trial in a Dish with Pooled iPSC-CMs for Drug Discovery
Translational Candidate Human stem cells in a dish engineered into heart cells to supplement, refine, reduce, and/or ultimately replace human clinical trials. Area of Impact Increase genetic diversity of preclinical studies in human samples to derisk clinical trials and save time and costs. Mechanism of Action We will have several non-invasive human-derived stem cells collected […]