Therapeutic/Technology: Assay development
A Chromatin Context Tool for Predicting iPS Lineage Predisposition and Tissue Graftability
Induced pluripotent stem (iPS) cells are cells derived from skin that closely resemble embryonic stem (ES) cells and can be coaxed into many different types of cells such as nerve cells, heart cells, liver cells, and also back to skin cells. One major bottleneck in the field is our ability to coax the cells into […]
User-friendly predictive molecular diagnostic assays for quality control of stem cell derivatives for transplantation and drug discovery
Three years ago, with help from CIRM funding, we developed an assay. This is a genomics-base diagnostic assay, similar to those now used for diagnosing cancers; but in our case, it is designed to analyze human ES and iPS cells. The assay is very simple to use; researchers use microarrays to profile the genes that […]
Center of Excellence for Stem Cell Genomics – UCSC
The Center of Excellence in Stem Cell Genomics will bring together investigators from seven major California research institutions to bridge two fields – genomics and pluripotent stem cell research. The projects will combine the strengths of the center team members, each of whom is a leader in one or both fields. The program directors have […]
Use of hiPSCs to develop lead compounds for the treatment of genetic diseases
This study will use Ataxia-Telangiectasia (A-T), an early-onset inherited neurodegenerative disease of children, as a model to study the mechanisms leading to cerebellar neurodegeneration and to develop a drug that can slow or halt neurodegeneration. We will start with skin cells that were originally grown from biopsies of patients with A-T who specifically carry “nonsense” […]
Crosstalk: Inflammation in Parkinson’s disease (PD) in a humanized in vitro model
Parkinson’s Disease (PD) is the most common neurodegenerative movement disorder. It is characterized by motor impairment such as slowness of movements, shaking and gait disturbances. Age is the most consistent risk factor for PD, and as we have an aging population, it is of upmost importance that we find therapies to limit the social, economic […]
Maximizing the Safety of Induced Pluripotent Stem Cells as an Infusion Therapy: Limiting the Mutagenic Threat of Retroelement Retrotransposition during iPSC Generation, Expansion and Differentiation
The ability to convert human skin cells to induced pluripotent stem cells (IPSCs) represents a seminal break-through in stem cell biology. This advance effectively circumvents the problem of immune rejection because the patient’s own skin cells can be used to produce iPSCs. This exciting technology could accelerate treatments for a number of presently incurable diseases. […]
Ensuring the safety of cell therapy: a quality control pipeline for cell purification and validation
The clinical application of cell replacement therapy in the US is dependent on the FDA’s approval, and the primary objective of the FDA is to protect patients from unsafe drugs and procedures. The FDA has a specific mandate for human gene and cell therapy and since the unexpected deaths in early trials of gene therapy […]
Methods for detection and elimination of residual human embryonic stem cells in a differentiated cell product
Human embryonic stem cells (hESC), and other related pluripotent stem cells, have great potential as starting material for the manufacture of curative cell therapies. This is primarily for two reasons. First, by manipulating cues in their cell culture conditions, these cells can be directed to become essentially any desired human cell type (a property known […]
Establishment of a novel approach to systematically study the dynamic organization of protein complexes in stem cells
Research Objective We focus on hiPSCs pluripotency and neurodifferentiation to develop a novel framework to allow simultaneous identification of multiple interactions between proteins and between proteins and the genome Impact Our framework will allow high-throughput queries of the organization and functionality of proteins and shift the focus towards unprecedented, multi-dimensional studies of the cellular complexity. […]