Program Type: Discovery
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. […]
Blood Brain Barrier (BBB)-on-Chip: Development and validation of a novel iPS-based microfluidic model of the human BBB
Research Objective to develop and systematically characterize a novel model of the human BBB using a microfluidic device (chip) and cells derived from induced pluripotent stem cells (iPSCs). Impact The success of the proposed research will provide a novel, highly attractive model for screening of molecules to treat neurological disorders and for personalized medicine in […]
Generation of bile duct-competent transplantable human liver organoids
Research Objective Generation of human stem cell-derived mini livers capable of exporting bile into the gallbladder after transplantation into the liver Impact Mini livers capable of normal bile export would have potential for therapy of diseases in which bile export is impaired like Alagille syndrome Major Proposed Activities Generation of mini livers using human stem […]
Targeting progenitors in scar tissue to reduce chronic scar burden
Research Objective Develop novel strategies to treat heart scars by targeting progenitors that replenish scars Impact There currently is no therapy for treating scar tissue in the heart or any other organ. Our proposal would lead to the development of targeted approaches to reduce scar burden. Major Proposed Activities Identify progenitors in scar tissue (murine […]
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 […]
Curing bladder cancer by replacing corrupted urothelium with differentiated hES cells
Research Objective The goal of the proposed research is to use human embryonic stem cells to generate bladder epithelial progenitor cells that can be used to replace a cancerous bladder epithelium in vivo. Impact The long-term goal of the proposed research is to cure bladder cancer. Successful completion of this work may indicate that transplantation […]
New Methods for the Chemical Expansion of Hematopoietic Stem and Progenitor Cells
Research Objective We will develop a new agent that can increase the production of hematopoietic stem and progenitor cells and determine how the compound functions Impact We aim to develop a method to achieve the highest fold expansion of hematopoietic stem cells from a single unit of cord blood achieved to date increasing the supply […]
Autologous iPSC-based therapy for radiation induced bladder injury
Research Objective To explore if iPSC-based therapy can prevent bladder damage due to radiation therapy, thereby limiting the unintended consequences of treatments for prostate, gynecologic and colorectal cancers. Impact This therapy impacts cancer survivors by preventing the permanent debilitating urinary symptoms due to radiation therapy. Currently there are no therapies to prevent radiation bladder damage. […]
iPS-Interneuron Transplantation for Neural Repair after Stroke
Research Objective To determine if transplantation of iPS-interneurons cells (iPS-3i cells) enhances functional recovery in stroke. Impact Successful completion of the proposed studies will develop a brain repair therapy for stroke, an unmet clinical need with significant impact on society. Major Proposed Activities To determine the recovery effect of transplantation of iPS-3i cells in the […]
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 […]