Project Objective: Tool/Resources/Bottleneck
A new precision medicine based iPSC-derived model to study personalized intestinal fibrosis treatments in pediatric patients with Crohn’s disease
Research Objective We propose to discover a tool that will utilize patient specific iPSC-derived human mini-guts to identify personalized antifibrotic treatments in pediatric Crohn’s disease patients Impact The major bottleneck in intestinal fibrosis research is the difficulty in obtaining patient-specific biologically relevant cells for in vitro modeling. This iPSC-derived tool would overcome it. Major Proposed […]
A screen for drugs to protect against chemotherapy-induced hearing loss, using sensory hair cells derived by direct lineage reprogramming from hiPSCs
Research Objective Development of a screen using inner ear sensory hair cell-like cells made by direct lineage reprogramming, for discovering drugs to ameliorate hearing loss during cancer chemotherapy. Impact Hearing loss, both adult and pediatric, due to life-saving cisplatin chemotherapies. Lack of human inner ear hair cells for drug discovery purposes and disease modeling. Major […]
Platform Technology for Pluripotent Stem Cell-Derived T cell Immunotherapy
Research Objective We will combine a novel method to produce T cells from stem cells with gene editing tools, to create pluripotent stem cells that can serve as a universal source of T cells for cancer immunotherapy. Impact We will address a major bottleneck for T cell immunotherapy: the complexity and therefore limited access to […]
Non-Toxic, Highly-Effective Bioinspired Cryoprotectants for On-Demand Stem Cell Therapies
Research Objective An advanced technology is sought to replace the toxic, ineffective and highly processed components in legacy cryopreservation media. This technology is a fundamentally novel non-toxic freezing media. Impact The transport and storage of stem cell therapies is crippled by freezing media with poor cell preservation. Significantly improved freezing media would directly increase therapy […]
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 […]
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 […]