Therapeutic/Technology: Technology
Ubiquitin-dependent control of hESC self-renewal and expansion
Human embryonic stem cells (hESCs) or induced pluripotent stem cells (iPSCs) provide an invaluable resource for regenerative medicine and disease modeling. To be able to use these cells in the clinic, hESCs and iPSCs need to be expanded without introducing genetic instability. However, current protocols of hESC and iPSC propagation frequently result in aneuploidy, a […]
Forming the Hematopoietic Niche from Human Pluripotent Stem Cells
The clinical potential of pluripotent stem cells for use in regenerative medicine will be realized only when the process by which tissues are generated from these cells is significantly more efficient and controlled than is currently the case. Fundamental questions remain about the mechanisms by which pluripotent stem cells differentiate into mature tissue. The overall […]
Biological relevance of microRNAs in hESC differentiation to endocrine pancreas
There remains an urgent and critical need for a cell-based cure of diabetes, one of the most costly diseases in California. Islet transplantation with persistent immune suppression has shown promise in curing type 1 diabetes (TID). However, one major obstacle towards large scale implementation of this approach is the shortage of engraftable islets. Human ES […]
Induced Pluripotent Stem Cells for Tissue Regeneration
Induced pluripotent stem cells (iPSCs) have tremendous potential for patient-specific cell therapies, which bypasses immune rejection issues and ethical concerns for embryonic stem cells (ESCs). However, to fully harness the therapeutic potential of iPSCs, many fundamental issues of cell transplantation remain to be addressed, e.g., how iPSC-derived cells participate in tissue regeneration, which type of […]
Homologous recombination in human pluripotent stem cells using adeno-associated virus.
Since their discovery in 1998, human embryonic stem cells (hESCs) have been considered to hold great potential for the treatment of many currently incurable diseases. Possibly the most exciting application of hESC in the clinic is in the arena of regenerative medicine where hESC-derived cell populations are used to replace diseased, damaged or dead tissues. […]
Cellular tools to study brain diseases affecting synaptic transmission
There is a group of brain diseases that are caused by functional abnormalities. The brains of patients afflicted with these diseases which include autism spectrum disorders, schizophrenia, depression, and mania and other psychiatric diseases have a normal appearance and show no structural changes. Neurons, the cellular units of the brain, function by making connections (or […]
Antibody tools to deplete or isolate teratogenic, cardiac, and blood stem cells from hESCs
Purity is as important for cell-based therapies as it is for treatments based on small-molecule drugs or biologics. Pluripotent stem cells possess two properties: they are capable of self regeneration and they can differentiate to all different tissue types (i.e. muscle, brain, heart, etc.). Despite the promise of pluripotent stem cells as a tool for […]
Tri-resolution Visualization System for Stem Cells and Tissue Regeneration Monitoring
The 3D imaging techniques of CT and MRI have virtually eliminated the need for exploratory surgery – a procedure which was common in difficult cases just 20-30 years ago. Not only is imaging used to discover the extent of disease, it is now used to measure the effect of therapies. The “size” of a tumor […]
Use of iPS cells (iPSCs) to develop novels tools for the treatment of spinal muscular atrophy.
Spinal Muscular Atrophy (SMA) is one of the most common lethal genetic diseases in children. One in thirty five people carry a mutation in a gene called survival of motor neurons 1 (SMN1) which is responsible for this disease. If two carriers have children together they have a one in four chance of having a […]
Engineering Defined and Scaleable Systems for Dopaminergic Neuron Differentiation of hPSCs
Human pluripotent stem cells (hPSC) have the capacity to differentiate into every cell in the adult body, and they are thus a highly promising source of differentiated cells for the investigation and treatment of numerous human diseases. For example, neurodegenerative disorders are an increasing healthcare problem that affect the lives of millions of Americans, and […]