Stage of Program: Basic Research and Discovery Stage Programs
Mitochondrial Metabolism in hESC and hiPSC Differentiation, Reprogramming, and Cancer
Stem cell quality and safety in developing regenerative medicine therapies is of utmost importance. Poor outcomes include inadequate functionality, exhaustion, immune rejection, cancer development, and others. Recent studies strongly support our core hypothesis that mitochondrial function determines stem cell quality and safety. Dysfunctional mitochondria foster cancer, diabetes, obesity, neurodegeneration, immunodeficiency, and cardiomyopathy. Unlike whole genome […]
The stem cell microenvironment in the maintenance of pluripotency and reprogramming
Pluripotent stem cell research is just on the verge of beginning to fulfill its promise to revolutionize medicine. Whether they are derived from embryos, or from adult cells that have been reprogrammed, human pluripotent stem cells can be propagated indefinitely in the laboratory and can turn into a wide range of mature cell types, providing […]
The retinoblastoma (RB) gene family in cellular reprogramming
One important aspect of regenerative medicine is the ability to introduce functional stem cells into patients to restore tissue function. This type of therapeutic approach will not be commonly used until several major potential problems have been addressed, including immune rejection and the risk of developing cancer. Induced pluripotent stem cells (iPSCs) hold great promise […]
Identification and characterization of human ES-derived DA neuronal subtypes
Parkinson’s disease (PD) is a neurodegenerative movement disorder that affects 1 in 100 people over the age of 60, one million people in the US and six million worldwide. Patients show a resting tremor, slowness of movement (bradykinesia), postural instability and rigidity. Parkinson’s disease results primarily from the loss of neurons deep in the middle […]
Molecular Characterization of hESC and hIPSC-Derived Spinal Motor Neurons
One of the main objectives of stem cell biology is to create physiologically relevant cell types that can be used to either facilitate the study of or directly treat human disease. Tremendous progress towards these goals has been made in the area of motor neuron disease and spinal cord injury through the findings that motor […]
Human Cardiovascular Progenitors, their Niches and Control of Self-renewal and Cell Fate
For the millions of Americans who are born with or develop heart disease, stem cell research offers the first hope of reversing or repairing heart muscle damage. Thus, early reports suggesting heart regeneration after transplantation of adult bone marrow-derived stem cells were met with great excitement in both the scientific and lay community. However, although […]
Defining the molecular mechanisms of somatic cell reprogramming
The development of methods to “reprogram” adult cells such as skin cells by simultaneously expressing four specific factors — Oct3/4, Sox2, c-Myc and Klf4 — in order to create cells resembling embryonic stem (ES) cells is a major breakthrough in stem cell biology. Our ability to generate these cells, which are known as induced pluripotent […]
Molecular Mechanisms of Reprogramming towards Pluripotency
Stem cell biology and its applications to cell-based therapies, since its inception 30 years ago, has been hindered by the immunological considerations of rejection of non-autologous cells in patients, as well as by ethical concerns. The generation of pluripotent cells from a patient’s own somatic cells has therefore been the holy grail of regenerative medicine. […]
Molecular Characterization and Functional Exploration of Hemogenic Endothelium
Hematopoietic cells are responsible for generating all cell types present in the blood and therefore critical for the provision of oxygen and nutrients to all the tissues in the body. Blood cells are also required for defense against microorganisms and even for the recognition and elimination of tumor cells. Because blood cells have a relatively […]
Deep phenotyping of human brain organoid models of autism spectrum disorder to unravel disease heterogeneity and develop biomarkers and treatments
Research Objective We will uncover pathways through which ASD mutations cause disease and close the gap from disease research to therapeutic testing using organoids, primary human neurons, machine learning and AAVs. Impact Our studies are impactful because outcomes will lead to therapeutic avenues to pursue for ASD treatment. 1 in 22 children in California is […]