Program Type: Discovery
Somatic cell age and memory in the generation of iPS cells
Pluripotent stem cells can give rise to any cell type of the body and hold enormous promise for regenerative medicine. Pluripotent stem cells, such as embryonic stem (ES) cells, are derived from very young human embryos. It is of great interest to derive pluripotent stem cells from adult cells. In this way, one could potentially […]
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 […]
Establishment of Frontotemporal Dementia Patient-Specific Induced Pluripotent Stem (iPS) Cell Lines with Defined Genetic Mutations
We propose to generate induced pluripotent stem (iPS) cells from skin cells derived from human subjects with frontotemporal dementia (FTD). FTD accounts for 15–20% of all dementia cases and, with newly identified genetic causes, is now recognized as the most common dementia in patients under 65 years of age. FTD patients suffer progressive neurodegeneration in […]
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 […]
New Cell Lines for Huntington’s Disease
Huntington’s disease (HD) is a devastating neurodegenerative disease with a 1/10,000 disease risk that always leads to death. These numbers do not fully reflect the large societal and familial cost of HD, which requires extensive caregiving and has a 50% chance of passing the mutation to the next generation. Current treatments treat some symptoms but […]
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 […]
An in vitro and in vivo comparison among three different human hepatic stem cell populations.
Because there is still considerable morbidity and mortality associated with the process of transplantation, and because more than a thousand people die each year while on the liver transplantation list, it is evident that improved and safer liver transplantation would be valuable, as would approaches that provide for an increased number of transplantations in a […]
Prospective isolation of hESC-derived hematopoietic and cardiomyocyte stem cells
The capacity of human embryonic stem cells (hESCs) to perpetuate themselves indefinitely in culture and to differentiate to all cell types of the body has lead to numerous studies that aim to isolate therapeutically relevant cells for the benefit of patients, and also to study how genetic diseases develop. However, hESCs can cause tumors called […]
Understanding hESC-based Hematopoiesis for Therapeutic Benefit
Hematopoietic stem cell transplantation is the treatment of choice for many hematologic malignancies, and it is used to treat an expanding number of congenital blood disorders. However, only ~30% of patients who can benefit from this treatment have a matched sibling that can serve as the ideal donor. While the national marrow donor program and […]