Stage of Program: Translational Tool/Bottleneck
Optimization of Human Embryonic Stem Cell Derivation Techniques and Production/Distribution of GMP-Grade Lines
The government has strict rules for producing cells that will be transplanted into patients. For example, these regulations discourage the use of animal products that could transmit diseases to humans. In this context, the high-quality and tightly regulated procedures that govern other cell-based therapies, e.g., bone marrow transplants, will be applied to regenerative-type clinical applications […]
Induction of pluripotent stem cells by small RNA-guided transcriptional activation
Embryonic stem cells have great potential in therapeutic use to replace diseased or damaged tissues because they have the unique capability of giving rise to any cell type of the body while perpetuating their own identity, even after repeated cell divisions. Recent advances in this area have resulted in a new way to generate stem […]
Development of Induced Pluripotent Stem Cells for Modeling Human Disease
Human embryonic stem cells (hESC) hold great promise in regenerative medicine and cell replacement therapies because of their unique ability to self-renew and their developmental potential to form all cell lineages in the body. Traditional techniques for generating hESC rely on surplus IVF embryos and are incompatible with the generation of genetically diverse, patient or […]
Generation of Pluripotent Cell Lines from Human Embryos
Human embryonic stem cells (hESCs) hold significant promise for regenerative medicine. In this application our goal is to derive hESC lines from pre-implantation embryos to generate a source of low passage lines that can be used in research and to develop the procedures required to generate a clinic grade cell-based product. In this application we […]
Protein transduction of transcription factors: a non-genetic approach to generate new pluripotent cell lines from human skin.
More than 100,000 patients await for organ transplants nationwide this year. The ground-breaking discovery of new pluripotent human stem cell lines (iPS) derived from skin fibroblasts using a core of 3-5 transcription factors opens the door to patient-derived pluripotent stem cells and new approaches to organ and tissue replacement. Patient-derived stem cells could have an […]
Preclinical Model for Labeling, Transplant, and In Vivo Imaging of Differentiated Human Embryonic Stem Cells
The derivation and culture of human embryonic stem cells has provided new possibilities for treatment of a wide variety of human diseases because these cells have the potential to help regenerate and repair many types of damaged tissue. Diseases for which such cell-based treatments may be helpful include obstructive renal disease, a disorder for which […]
Patient-specific cells with nuclear transfer
Somatic cell nuclear transfer (NT) is a powerful research tool with the potential for creating unique cell and tissue sources for studies of disease pathogenesis and regenerative medicine. Creation of pluripotent mouse embryonic stem (ES) cells using NT has been achieved and the prospects for generating human ES cells by NT are promising. However, there […]
In Vivo Imaging of Human Embryonic Stem Cell Derivatives and Tumorigenicity
Human embryonic stem cells (hESCs) are one of the most fascinating subjects of interest in all of biology and medicine these days. Under certain physiologic conditions, they can be induced to become specialized cells such as brain, cardiac, liver, pancreatic, and bone marrow cells. This opens up the exciting possibility that hESCs may one day […]
Development of human ES cell lines as a model system for Alzheimer disease drug discovery
Alzheimer disease (AD) is a progressive neurodegenerative disorder that currently affects over 4.5 million Americans. By the middle of the century, the prevalence of AD in the USA is projected to almost quadruple. As current therapies do not abate the underlying disease process, it is very likely that AD will continue to be a clinical, […]
Novel vectors for gene transfer into human ES cells
Human embryonic stem cells have a great potential for medical therapeutics. However, the genes required for altering the fate of these cells to differentiate into a particular tissue or cell type is not well understood. The ability to efficiently transfer genes or silence genes in ES cells would be of great benefit for two reasons: […]