Stage of Program: Basic Research and Discovery Stage Programs


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 […]

hPSC-derived enteric ganglioids for cell therapy in gastrointestinal motility disorders

Research Objective The proposed aims will enable the generation, purification and characterization of enteric neurons from diverse hiPSCs and assessment of their efficacy for cell therapy in GI motility disorders. Impact This proposal addresses a significant unmet clinical need for a cell therapy approach for gastrointestinal motility disorders such as Hirschsprung disease, achalasia and gastroparesis. […]

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 […]

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 […]

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 […]

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 […]

Mapping the spatial and temporal responses of hESC-derived microglia to repeat mild closed head injury to identify therapeutic targets and mechanisms

Research Objective We will generate an RNA activation map of human stem cell derived microglia activation states following brain injury to then test a new gene-edited microglia peptide delivery mechanism. Impact Bottlenecks with the time and sex-dependent human microglia responses to repeat mild closed head injury and questions surrounding the delivery and efficacy of a […]

Role of ataxin-3 polyadenylation site selection in ALS neuron toxicity and disease pathogenesis

Research Objective Here we will study the role of ataxin-3 alternative polyadenylation in the pathogenesis of ALS, and test if ASOs can reduce distal polyadenylation of ataxin-3 to rescue ALS disease phenotypes Impact Our goal is to determine if ataxin-3 genetic dysregulation is a target for the development of therapies to treat ALS (Lou Gehrig's […]