Human Stem Cell Use: iPS Cell
Developing a personalized approach to beta cell replacement for patients with a genetic form of diabetes
Research Objective To correct a gene mutation in a patient's stem cells and produce functional replacement cells for the patient to cure their diabetes. Impact WE expect that this project can serve as a model for developing new treatments for patients with certain forms of genetic diabetes. Major Proposed Activities To understand how the patient's […]
Modulating Liver Sinusoidal Endothelial Cell Permeability to Enhance Engraftment of Endothelial Cell Progenitors for the Treatment of Hemophilia A
Research Objective We aim to demonstrate that regulators of endothelial cell permeability can foster engraftment of endothelial cell progenitors in the liver sinusoids leading to production of Factor VIII. Impact Our work would provide conceptual proof that a cell based therapy for hemophilia A is possible and should be pursued. Major Proposed Activities Demonstrate that […]
Embryonic Stem Cells for Corneal Endothelial Degeneration
Research Objective The proposed studies will determine the optimal approaches to differentiate and transplant stem cell-derived corneal endothelial cells. Impact These data will provide foundational proof-of-concept data that will allow the rapid advance of a cell therapy towards clinical application. Major Proposed Activities Determine optimal conditions to generate human corneal endothelial cells from human stem […]
Reverse transcriptase inhibitors as a novel therapeutic approach for neurological autoimmune disorders
Research Objective We found that approved anti-retroviral drugs could stop inflammation and block neurodegeneration. We propose to validate the re-purpose efficacy of these clinically-approved retroviral drugs. Impact We have identified an unexpected cause to a brain inflammation and a potential simple treatment. Our research could help millions of patients affected by a broad range neuro-immunological […]
Novel Platforms to Enhance In Vivo Delivery of Skeletal Muscle Progenitor Cells from Human Pluripotent Stem Cells
Research Objective Delivery of muscle stem cells presents a major roadblock for therapy. We explore novel approaches to increase the efficiency of delivering and monitoring muscle stem cells derived from hPSCs. Impact Development of enhanced monitoring and delivery platforms will greatly accelerate translational strategies aimed at delivering muscle stem cells for transplantation to patients with […]
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 […]
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 […]