Therapeutic/Technology: Technology
Elucidating Molecular Basis of Hypertrophic Cardiomyopathy with Human Induced Pluripotent Stem Cells
Familial hypertrophic cardiomyopathy (HCM) is the leading cause of sudden cardiac death in young people, including trained athletes, and is the most common inherited heart defect. Until now, studies in humans with HCM have been limited by a variety of factors, including variable environmental stimuli which may differ between individuals (e.g., diet, exercise, and lifestyle), […]
Characterization and Engineering of the Cardiac Stem Cell Niche
Despite therapeutic advances, cardiovascular disease remains a leading cause of mortality and morbidity in both California and Europe. New insights into disease pathology, models to expedite in vitro testing and regenerative therapies would have an enormous societal and financial impact. Although very promising, practical application of pluripotent stem cells or their derivatives face a number […]
USP16 controls stem cell number: implications for Down Syndrome
Stem cells are endowed with the ability to self-renew, that means to give rise to other cells with the same potential to regenerate a tissue. Recently, we found a gene that also regulates this mechanism. In addition, expression of high levels of this gene can reduce the number of stem cells in the bone marrow […]
Genomic instability during culturing of human embryonic stem cells
Human embryonic stem cells (hESCs) have important potential in the treatment of human disease. Because they can change into a large number of different cell types, they may be useful in restoring a variety of damaged tissues. One potentially harmful side effect of hESC therapy is cancer due to unregulated growth of the hESCs introduced […]
Generation and characterization of corticospinal neurons from human embryonic stem cells
A major goal of stem cell research is to generate various functional human cell types that can be used to better understand how these cells work and to use them directly in therapies. There are currently no effective treatments, let alone a cure, for many neurological conditions. Two particular devastating neurological conditions, spinal cord injury […]
Enhancer-mediated gene regulation during early human embryonic development
Less than 2% of the human genome encodes protein coding genes. But many trait-specific and disease specific mutations seem to map away from such coding sequences. This paradox is partially resolved by observation that some of the noncoding sequences are involved in regulation of when and where in the developing organism genes are to be […]
Molecular Mechanisms Underlying Human Cardiac Cell Junction Maturation and Disease Using Human iPSC
Heart disease is the number one cause of death and disability in California and in the United States. Especially devastating is Arrhythmogenic Right Ventricular Cardiomyopathy (ARVC), an inherited form of heart disease associated with a high frequency of arrhythmias and sudden cardiac death in young people, including young athletes, who despite their appearance of health […]
Molecular determinants of accurate differentiation from human pluripotent stem cells
The use of human pluripotent stem cells for cell-based therapeutics is predicated on the ability to convert these cells into functional equivalents of those lost in disease or injury. However, there is only scant evidence that either human embryonic stem cells or human induced pluripotent stem cells make differentiated progeny that are functionally equivalent to […]
Etsrp/ER71 mediated stem cell differentiation into vascular lineage
Human embryonic stem cells (hESC) have the potential to differentiate into all of the cell types that make up the body. Therefore, hESCs are promising tools for the treatment of degenerative diseases and for use in regenerative medicine. One highly desirable use of hESCs is to treat cardiovascular disease. Cardiovascular disease is a leading cause […]
Understanding the role of LRRK2 in iPSC cell models of Parkinson’s Disease
The goal of this research is to utilize novel research tools to investigate the molecular mechanisms that cause Parkinson’s disease (PD). The proposed work builds on previous funding from CIRM that directed the developed patient derived models of PD. The majority of PD patients suffer from sporadic disease with no clear etiology. However some PD […]