Therapeutic/Technology: Assay development

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. […]

Safety “Clinical Trial” of the Cardiac Liability of COVID19 Polytherapy

Global gene expression profiling of single inhibitory neurons derived from human stem cells

Autologous therapy for Parkinson’s disease: single cell RNAseq for in depth characterization of transplanted cells

A screen for drugs to protect against chemotherapy-induced hearing loss, using sensory hair cells derived by direct lineage reprogramming from hiPSCs

Research Objective Development of a screen using inner ear sensory hair cell-like cells made by direct lineage reprogramming, for discovering drugs to ameliorate hearing loss during cancer chemotherapy. Impact Hearing loss, both adult and pediatric, due to life-saving cisplatin chemotherapies. Lack of human inner ear hair cells for drug discovery purposes and disease modeling. Major […]

A Novel, Robust and Comprehensive Predictive Tool Using Human Disease-Specific Induced Pluripotent Stem Cells for Preclinical Drug Screening

Translational Candidate A library of induced pluripotent stem cell-derived cardiomyocytes from healthy subjects as well as patients with common hereditary cardiac disorders Area of Impact Preclinical toxicity screening and drug discovery Mechanism of Action Patients with pre-existing cardiac conditions are more susceptible to drug-induced cardiotoxicity than general population. Including iPSCs derived from this subset of […]

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” […]