Human Stem Cell Use: Adult or Tissue Stem Cell
Stem Cell-based Disease Modeling Shared Resource Laboratory
Our Shared Resource Laboratory will expand access to stem cell-derived models to support research and educational programs with two main goals: democratize access to state-of-the-art models and technology among a diverse scientific community and catalyze innovation in regenerative medicine. This Shared Resource Laboratory will expand access to stem cell-derived models to regions of Southern California […]
Harnessing the rejuvenating capacity of pregnancy-associated factors to restore aged stem cell function
Research Objective Elucidation of pregnancy-related factors that mitigate cellular senescence and enhance regeneration has far-reaching implications for understanding the mechanisms of aging and rejuvenation. Impact The study will address the long-standing knowledge gaps related to the mechanisms of pro-regenerative impact of pregnant milieu and female muscle stem cell senescence Major Proposed Activities Isolation of female […]
Interrogating Satellite Cell and Myofiber Defects and Repair in Human DMD using Single Nuclei/Single Cell RNA Sequencing of Muscle Resident Cells
Research Objective We will describe, for the first time, human muscle satellite cell, myofiber and immune cell dynamics due to dystrophin deficiency and AAV gene therapy in human muscle at single nuclei resolution. Impact These studies will elucidate satellite stem cell and myofiber defects in Duchenne and Becker Muscular Dystrophy and determine efficacy, mechanism and […]
Utilizing Age-Specific Adipocyte Progenitor Cells for Cell Therapy in Older Patients
Research Objective A new type of APC serves older patients as 1) better MSC in immunomodulation (reducing inflammation) for autologous transplantation; 2) better source of somatic cells for generating heathier hiPSCs. Impact Bottlenecks: 1) Older patients suffer from sarcopenic obesity, which has no safe and effective treatment. 2) Cell therapy in older patients is often […]
Ex vivo fate mapping of human lung stem cell plasticity in fibrotic disease
Research Objective This proposal will design new models to study behavior that is specific to human lung stem cells, and screen for drugs that can target abnormal stem cells in fibrotic disease. Impact This study will generate potential new therapeutic approaches to fibrotic lung diseases such as idiopathic pulmonary fibrosis. Major Proposed Activities Development of […]
Overcoming barriers for airway stem cell gene therapy for Cystic Fibrosis
Research Objective This research will allow the targeting of airway stem cells for long lived gene therapy for Cystic Fibrosis and for other airway diseases Impact We will overcome the barriers to accessing airway basal stem cells for gene correction for Cystic Fibrosis (CF) and use a new gene correction strategy to correct >99% of […]
Identifying roadblocks to neural stem cell transplantation into human tissues.
Research Objective We will generate a comprehensive map of human neural stem cell differentiation profiles that will serve as a reference for enhancing neural stem cell-based therapies. Impact Our project will develop improved protocols for human neural stem cells differentiation, enhancing the fidelity, safety and robustness of future cell therapies. Major Proposed Activities Establish quantitative […]
Determining how age-specific heterogeneity of human hematopoietic stem cells and megakaryocyte progenitors contribute to thrombotic disease upon aging
Research Objective Our research will determine how aging of human blood stem cells leads to dramatic increases in disorders of platelets, cells that normally prevent bleeding but form harmful clots when dysregulated. Impact Our findings have the potential to inform prevention and mitigation strategies of bleeding and clotting disorders that contribute to significant morbidity and […]
Harnessing vascular stem cells to grow and protect the human brain
Research Objective The origins of brain vascular mural cells are unknown. This proposal will identify mural stem cells in the developing human and mouse brain and determine their impact on blood brain barrier formation. Impact Brain vascular diseases can have profound impacts on long-term neurological function. This proposal will map the stages of mural stem […]
Modulation of human alveolar stem cells to promote lung regeneration and avoid pulmonary fibrosis
Research Objective Understanding regulators of human alveolar lung stem cell function will promote more normal lung regeneration after injury and avoid the nearly untreatable problem of advanced pulmonary fibrosis. Impact Idiopathic Pulmonary Fibrosis (IPF), Adult Respiratory Distress Syndrome (ARDS), and other chronic fibrotic lung disorders. Major Proposed Activities Modify human fibroblasts to create a supporting […]