Project Objective: Proof of Concept

iPSC-Derived Smooth Muscle Progenitors for Treatment of Abdominal Aortic Aneurysm

Research Objective To assess the therapeutic effect of human induced pluripotent stem cell (iPSC)-derived smooth muscle progenitors (pSMCs) for treatment of abominal aortic aneurysm (AAA). Impact Currently, there are no pharmacologic therapies for AAA. If successful, delivery of autologous pSMCs to the site of AAA will halt or reverse the progression towards a rupture-prone aneurysm. […]

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 […]

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 […]

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. […]

Hypo-immunogenic cardiac patches for myocardial regeneration

Research Objective To engineer a cardiac patch to restore function after a heart attack while avoiding an immune response (“hypo-immunogeneic” CP) when transplanted into a genetically distinct (“allogenic”) individual. Impact By making hypo-immunogeneic CPs and functional cardiac cells (induced pluripotent stem, “iPS” cells) available to commercial/research entities, our study can fuel the transformation of healthcare. […]

A Novel Tissue Engineering Technique to Repair Degenerated Retina

Research Objective Transplantation of human embryonic stem cell (hESC) derived retina organoids (hESC-RO) together with hESC derived retinal pigment epithelium (hESC-RPE) to treat advanced retinal degeneration diseases Impact Based on the ‘proof of concept’ experiments in animal models, this novel approach can be translated into a therapeutic product for the treatment of advanced human retinal […]

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 […]