Therapeutic/Technology: Technology
Investigating the SGF29/SAGA complex in regulation of normal and cancer stem cells
Research Objective This study will address gaps in our understanding of how normal and cancer stem cells differ in their epigenetic states, helping develop new cancer-stem-cell-targeting therapis. Impact The long-term impact of our studies is the understanding of mechnistic differences between normal and cancer stem cells and the development of new therapies. Major Proposed Activities […]
Village-based identification of human risk factors for viral neuropathogenesis
Research Objective We will identify the risk factors underlying viral infections of the fetal brain using a novel human stem cell-based platform that has the potential to accelerate basic and translational discoveries. Impact Our work will identify the biological factors that influence inter-individual differences in susceptibility and immune response to neurotropic viruses, which could inform […]
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 […]
Modeling and understanding alveolar hypoplasia in Down syndrome using iPSCs-derived alveolar type II cells
Research Objective Understanding alveolar progenitor cell defects in T21 and the genes/pathways associated with them will allow for developing therapeutic approaches for individuals with DS. Impact Although trisomy 21 affects multiple organ system, respiratory complications are the major cause of death in kids and adults with DS. The causes of lung disease in DS remain […]
Defining the source of dysfunction in monogenic Intellectual Disability Syndrome neurons
Research Objective This study will use pluripotent stem cells derived from patients to determine why Intellectual Disabilities caused by mutations in chromatin regulatory proteins leads to neuronal defects. Impact Our study of intellectual disability syndromes will determine links between mutations and neuronal dysfunction Major Proposed Activities Our study of intellectual disability syndromes will determine links […]
Characterization and applications of human blastoids for understanding early human embryogenesis
Research Objective Our work will yield an improved stem-cell based embryo model that we will explore with various omics approaches and genetic screens to gain insights into the pathways that control human embryos. Impact An improved stem cell-based embryo model is a crucial step for in-depth studies of human development and will enhance our ability […]
An interactive data resource for hypothesis testing in stem cell single-cell gene expression and validation of the results with brain organoids
Research Objective We are building a "virtual molecular microscope" where anyone can quickly visualize a very recent, high-throughput molecular assay, single-cell RNA-seq and spatial gene expression studies Impact Currently, a lot of data has been published, hundreds of datasets on the cerebral cortex alone, but it takes hours to convert the datasets and look at […]
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 […]
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 […]
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 […]