Program Type: Discovery


Trophoblast differentiation of human ES cells.

Human embryonic stem (ES) cells have the potential to form any cell type, but ironically, the first cell lineage to form during development still represents a surprising challenge. The first cell type to become specialized is an epithelial cell that later defines the boundary between the embryo and mother for the formation of the placenta. […]

Gene regulatory mechanisms that control spinal neuron differentiation from hES cells.

More than 600 disorders afflict the nervous system. Common disorders such as stroke, epilepsy, Parkinson’s disease and autism are well-known. Many other neurological disorders are rare, known only to the patients and families affected, their doctors and scientists who look to rare disorders for clues to a general understanding of the brain as well as […]

Modeling Human Embryonic Development with Human Embryonic Stem Cells

Stem cells have entered the public consciousness as “cells that can do anything” and have been hailed as a panacea in the fight against disease, aging and cancer. Unfortunately, we have only scratched the surface in understanding these cells. Some of the things we think we know are that: embryonic stem cells hold great promise […]

Development of human ES cell lines as a model system for Alzheimer disease drug discovery

Alzheimer disease (AD) is a progressive neurodegenerative disorder that currently affects over 4.5 million Americans. By the middle of the century, the prevalence of AD in the USA is projected to almost quadruple. As current therapies do not abate the underlying disease process, it is very likely that AD will continue to be a clinical, […]

Cellular epigenetic diversity as a blueprint for defining the identity and functional potential of human embryonic stem cells

Human embryonic stem (ES) cells have the capacity to self-renew but also give rise to other cell types. How this capacity is regulated and what factors determine one fate over another is an active area of research. This is because by understanding the decision making process the a stem cell goes through, we might be […]

Novel vectors for gene transfer into human ES cells

Human embryonic stem cells have a great potential for medical therapeutics. However, the genes required for altering the fate of these cells to differentiate into a particular tissue or cell type is not well understood. The ability to efficiently transfer genes or silence genes in ES cells would be of great benefit for two reasons: […]

New Chemokine-Derived Therapeutics Targeting Stem Cell Migration

This proposal describes a sharply-focused, timely, and rigorous effort to develop new therapies for the treatment of injuries of the Central Nervous System (CNS). The underlying hypothesis for this proposal is that chemokines and their receptors (particularly those involved in inflammatory cascades) actually play important roles in mediating the directed migration of human neural stem […]

Therapeutic potential of Retinal Pigment Epithelial cell lines derived from hES cells for retinal degeneration.

Retinal degeneration represents a group of blinding diseases that are increasingly impacting the health and well being of Californians. It is estimated that by 2020, over 450,000 Californians will suffer from vision loss or blindness due to the age-related macular degeneration (AMD), the most common cause of retinal degeneration diseases in the elderly. Furthermore, retinitis […]

Optimization of guidance response in human embryonic stem cell derived midbrain dopaminergic neurons in development and disease

A promising approach to alleviating the symptoms of Parkinson’s disease is to transplant healthy dopaminergic neurons into the brains of these patients. Due to the large number of transplant neurons required for each patient and the difficulty in obtaining these neurons from human tissue, the most viable transplantation strategy will utilize not fetal dopaminergic neurons […]

The APOBEC3 Gene Family as Guardians of Genome Stability in Human Embryonic Stem Cells

The successful use of human embryonic stem cells (hESCs) as novel regenerative therapies for a spectrum of currently incurable diseases critically depends upon the safety of such cell transfers. hESCs contain roughly 3 million “jumping genes” or mobile genetic retroelements that comprise up to 45% of their genetic material. While many of these retroelements have […]