Human Stem Cell Use: Embryonic Stem Cell
Development of Suspension Adaptation, Scale-up cGMP Banking and Cell Characterization Technologies for hESC Lines
hESCs represent an important source of cell therapies in regenerative medicine and the study of early human development. A number of hESC-based therapies are nearing clinical trials. To bring these to clinical trials requires the scale-up production, or “banking”, of large numbers of the desired hESC cell. The current lack of large scale hESC culture […]
Optimization in the Identification, Selection and Induction of Maturation of Subtypes of Cardiomyocytes derived from Human Embryonic Stem Cells
Cardiovascular diseases remain the major cause of death in the western world. Stem and progenitor cell-derived cardiomyocytes (SPC-CMs) hold great promise for the myocardial repair. However, most of SPC-CMs displayed heterogeneous and immature electrophysiological phenotypes with substantial automaticity. Implanting these electrically immature and inhomogeneous CMs to the hearts would be arrhythmogenic and deleterious. Further optimization […]
The Stem Cell Matrix: a map of the molecular pathways that define pluripotent cells
Human embryonic stem cells (hESC) are being considered for a wide range of research and therapeutic uses. Cell therapy is the most challenging of the potential clinical applications and its success will depend on the ability to guide differentiation of hESC into clinically useful cell types. The ideal cell types would possess three features: the […]
Development of a novel technology for precise, efficient, and safe genetic modification of stem cells
Stem cells are unique among cell types found in the human body: These cells are pluripotent; that is, they can develop into any of the more than 200 cell types in the human body. A major goal of stem cell research is to develop treatments for patients who suffer from devastating and currently incurable conditions […]
TAT Cell-Permeable Protein Delivery of siRNAs for Epigenetic Programming of Human Pluripotent and Adult Stem Cells
The rapid progress of embryonic stem cell, induced-pluripotent cell, and adult stem cell research opens the door to thousands of promising, new medical applications and discoveries. However, one of the major obstacles in translating these basic science discoveries into safe therapies for patients is the risk of acquiring mutations from viral and DNA vectors. Exposure […]
Synthetic Matrices for Stem Cell Growth and Differentiation
There is a critical need for new technologies to facilitate growth and differentiation of human embryonic stem cells (hESC) using clinically acceptable, animal-free reagents. In particular, most currently used culture conditions are not acceptable for standardized production of clinical grade cell products. We propose to develop novel, well-defined, synthetic extracellular matrices for growth and differentiation […]
Scaleup of Versatile, Fully Automated, Microfluidic Cell Culture System
We are proposing to optimize and scale up a highly advanced (microfluidic) cell culture system into manufacturable form. This system will allow researchers to: Identify stem cell culture and differentiation conditions Identify genes and small molecules effecting stem cell self-renewal and differentiation, and Identify genes and small molecules involving or effecting reprogramming of differentiated cells. […]
Microfluidic Platform for Screening Chemically Defined Conditions that Facilitate Clonal Expansion of Human Pluripotent Stem Cells
Human pluripotent stem cells (hPSCs) hold a great potential to treat many devastating injuries and diseases. However, current hPSC cloning still faces challenges in creating animal product-free culture conditions for performing genetic manipulation and induced differentiation of hPSCs for cell-based therapy. In order to obtain the ideal culture conditions for hPSC cloning, microfluidic technology can […]
Development of an hES Cell-Based Assay System for Hepatocyte Differentiation Studies and Predictive Toxicology Drug Screening
Drug-induced liver toxicity, including that from FDA-approved drugs, is the leading cause of liver failure in the US. One of the biggest road blocks to testing drug-induced liver toxicity prior to clinical studies or release of the drug into the market is the absence of a good model of human drug metabolism in the liver. […]
Directed Evolution of Novel AAV Variants for Enhanced Gene Targeting in Pluripotent Human Stem Cells and Investigation of Dopaminergic Neuron Differentiation
Human embryonic stem cells (hESCs) and induced pluripotent stem (iPS) cells have considerable potential as sources of differentiated cells for numerous biomedical applications. The ability to introduce targeted changes into the DNA of these cells – a process known as gene targeting – would have very broad implications. For example, mutations could readily be introduced […]