Stage of Program: Basic Research and Discovery Stage Programs
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 […]
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 […]
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 […]
Genetic Enhancement of the Immune Response to Melanoma via hESC-derived T cells
The overall goal of the proposed studies is to utilize human gene therapy approach using human embryonic stem cells to direct our body’s defenses to specifically attack melanoma tumor cells. Current technologies try to accomplish this by genetically manipulating certain circulating T lymphocytes, such that they will target tumor cells. T lymphocytes are the major […]
Generation of forebrain neurons from human embryonic stem cells
The goal of this proposal is to generate forebrain neurons from human embryonic stem cells. Our general strategy is to sequentially expose ES cells to signals that lead to differentiation along a neuronal lineage, and to select for cells that display characteristics of forebrain neurons. These cells would then be used in transplantation experiments to […]
Genetic modification of the human genome to resist HIV-1 infection and/or disease progression
The proposed studies describe the genetic approaches utilizing human embryonic stem cells to suppress and/or eliminate the expression of the human protein CCR5. CCR5 is found on the surface of white blood cells. HIV-1 attaches to CCR5 and uses CCR5 to enter into its target cells. Our approach is to utilize established as well as […]
Role of Mitochondria in Self-Renewal Versus Differentiation of Human Embryonic Stem Cells
Human embryonic stem cells (hESCs) hold great potential for treating multiple human dread diseases, including but not limited to cancer, diabetes, obesity, Alzheimer disease, and certain types of heart failure. However, a growing appreciation exists for the notion that not all hESCs have identical capabilities in correcting or ameliorating disease and not all hESCs will […]