Project Objective: Proof of Concept
A new phenotypic screening platform that identifies biologically-relevant targets and lead compounds for the treatment of Parkinson’s disease
Research Objective Demonstrate that our HitFinder™ library can be screened for phenotypic changes in A53T-IPSC-derived dopaminergic neurons and use a secondary handle to identify the targets responsible. Impact This technology combines phenotypic screening and target-ID eliminating the need to bias assays and/or screening libraries permitting application directly in iPSC-derived cells. Major Proposed Activities Prepare screening […]
IVD rejuvenation using iPSC-derived notochordal cells
Research Objective To identify a new therapeutic agent for disc regeneration using novel pluripotent stem cells and injectable beads that support differentiation and provide biomechanical strength. Impact If this study is successful, we will be able to bring completely new biologically and biomechanically relevant solution to degenerated intervertebral discs. Major Proposed Activities To optimize stem […]
Bone Marrow Targeting of Hematopoietic Stem Cells Engineered to Overexpress 25-OH-VD3 1-α-hydroxylase for Acute Myeloid Leukemia Therapy
Research Objective We propose a new approach to differentiation therapy for acute myeloid leukemia by producing local level of high-dose vitamin D in bone marrow via cell therapy with engineered hematopoietic stem cells Impact If proven successful, the proposed research can serve as a major breakthrough in the treatment of multiple subtypes of AML and […]
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 […]