Stage of Program: Candidate Discovery (DISC2, some 1.0 projects)
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 […]
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 […]
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 […]
Using human embryonic stem cells to treat radiation-induced stem cell loss: Benefits vs cancer risk
A variety of stem cells exist in humans throughout life and maintain their ability to divide and change into multiple cell types. Different types of adult derived stem cells occur throughout the body, and reside within specific tissues that serve as a reserve pool of cells that can replenish other cells lost due to aging, […]
In Vivo Molecular Magnetic Resonance Imaging of Human Embryonic Stem Cells in Murine Model of Myocardial Infarction
Magnetic resonance imaging (MRI) has emerged as one of the predominant modalities to evaluate the effects of stem cells in restoring the injured myocardium. However, MRI does not enable assessment of a fundamental issue in cell therapy, survival of the transplanted cells. The transplanted human embryonic cells (hESC) must at the very least survive to […]
Generation of long-term cultures of human hematopoietic multipotent progenitors from embryonic stem cells
For many therapeutic reasons it is important to have available large numbers of blood cells. However, it is difficult to generate large numbers of specialized blood cells that have the ability to neutralize autoimmunity and response to tumor cell growth. In this study we would develop a technique that would allow the production of large […]
Micro Platform for Controlled Cardiac Myocyte Differentiation
Congestive heart failure, the inability of the heart to continue to pump effectively due to damage of its muscle cells, affects approximately 4.8 million Americans and is a leading cause of mortality. Causes of the irreversible damage to the cardiomyocytes that results in congestive heart failure include hypertension, heart attacks, and coronary disease. Because the […]
Micro Platform for Controlled Cardiac Myocyte Differentiation
Congestive heart failure, the inability of the heart to continue to pump effectively due to damage of its muscle cells, affects approximately 4.8 million Americans and is a leading cause of mortality. Causes of the irreversible damage to the cardiomyocytes that results in congestive heart failure include hypertension, heart attacks, and coronary disease. Because the […]
Modeling Retinitis Pigmentosa using patient-derived human iPSC organoids
Research Objective The objective of this proposal is to develop a human retinal organoid model of adRP to gain insights in pathogenesis and assess clinically relevant approaches to restore RHO protein function. Impact Upon successful completion of this study, we will have established a disease-in-a-dish model and a novel therapeutic approach towards management of the […]