Therapeutic/Technology: Model system development

Blood Brain Barrier (BBB)-on-Chip: Development and validation of a novel iPS-based microfluidic model of the human BBB

Research Objective to develop and systematically characterize a novel model of the human BBB using a microfluidic device (chip) and cells derived from induced pluripotent stem cells (iPSCs). Impact The success of the proposed research will provide a novel, highly attractive model for screening of molecules to treat neurological disorders and for personalized medicine in […]

Genome editing for causation and reversion of MPN-associated mutations in human hematopoietic stem cells

Research Objective Use gene editing to create tools for the study of mechanisms by which patient-observed mutations lead to myeloproliferative neoplasms. Impact Editing reagents will yield new insight into how acquired MPN-associated mutations cause disease by overproduction of various cell types and pave the way for gene editing therapies to reverse MPNs. Major Proposed Activities […]

Cell Villages and Clinical Trial in a Dish with Pooled iPSC-CMs for Drug Discovery

Translational Candidate Human stem cells in a dish engineered into heart cells to supplement, refine, reduce, and/or ultimately replace human clinical trials. Area of Impact Increase genetic diversity of preclinical studies in human samples to derisk clinical trials and save time and costs. Mechanism of Action We will have several non-invasive human-derived stem cells collected […]

Human iPSC-derived micro-heart muscles for high-throughput cardiac drug discovery

Translational Candidate In vitro miniaturized array of heart muscle amenable for use in efficient high-throughput drug discovery and screening campaigns. Area of Impact Effective high-throughput screening of drugs on human heart muscles does not exist, hindering the discovery of therapeutics to treat heart failure. Mechanism of Action Current approaches for drug discovery often miss a […]

Embryonic stem cell-based generation of rat models for assessing human cellular therapies

Heart failure, diabetes and neurodegenerative diseases are among the leading causes of death and disability worldwide. These diseases are characterized by the loss of specific cell types and can be treated and potentially cured with stem cell-based therapies. Before human stem cells can be used in clinical trials, however, their safety and efficacy need to […]

Macaca mulatta as advanced model for predictive preclinical testing of engineered cardiac autografts and allografts

Heart disease is the number one cause of death in the US. Heart muscle injured during a heart attack does not regenerate, and the resulting damage leads to heart failure, which inflicts almost 6 million people in the US alone. Recently, several studies have shown that direct injection of stem cell-derived heart cells may offer […]

Development of a Relevant Pre-Clinical Animal Model as a Tool to Evaluate Human Stem Cell-Derived Replacement Therapies for Motor Neuron Injuries and Degenerative Diseases

Motor neurons degenerate and die as a consequence of many conditions, including trauma to the spinal cord and its nerve roots and degenerative diseases such as amyotrophic lateral sclerosis and spinal muscular atrophy. Paralysis and in many cases death may result from a loss of motor neurons. No effective treatments are available for these patients. […]

Center of Excellence for Stem Cell Genomics – Stanford

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 […]

In vitro modeling of human motor neuron disease

Motor neuron (MN) diseases such as spinal muscular atrophy and amyotrophic lateral sclerosis lead to progressive degeneration of MNs, presenting first with muscle weakness, followed by locomotor defects and frequently death due to respiratory failure. While progress has been made in identifying genes associated with MN degeneration, the molecular and cellular processes underlying disease onset […]

Misregulated Mitophagy in Parkinsonian Neurodegeneration

Parkinson’s disease (PD), is one of the leading causes of disabilities and death and afflicting millions of people worldwide. Effective treatments are desperately needed but the underlying molecular and cellular mechanisms of Parkinson’s destructive path are poorly understood. Mitochondria are cell’s power plants that provide almost all the energy a cell needs. When these cellular […]