Therapeutic/Technology: Exploring stem cell mechanisms

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 […]

Identifying sources of mutation in human induced pluripotent stem cells by whole genome sequencing

Stem cell research offers new tools to help treat and cure diseases that affect diverse cells types in the body such as neurological diseases, heart disease and diabetes by producing human cells for transplantation or to enable drug discovery . Recent advances have allowed researchers to generate patient-matched cell types from the skin or other […]

Ubiquitin-dependent control of hESC self-renewal and expansion

Human embryonic stem cells (hESCs) or induced pluripotent stem cells (iPSCs) provide an invaluable resource for regenerative medicine and disease modeling. To be able to use these cells in the clinic, hESCs and iPSCs need to be expanded without introducing genetic instability. However, current protocols of hESC and iPSC propagation frequently result in aneuploidy, a […]