Multi-gene modulation to rescue CNS-associated microdeletion syndromes
Research Objective Chromosomal microdeletion syndromes result in severe neuropsychiatric syndromes and lack therapy. This proposal will define critical genomic regions needed to generate new tools for functional rescue. Impact This…
Global profiling of miRNA-based gene activation to enable a new category of genetic medicine
Research Objective The proposed research will address key knowledge gaps in small activating RNAs (saRNAs) and could overcome bottlenecks limiting the broader adoption of this technology for regenerative medicine Impact…
Dissecting the cellular and molecular interactions between embryo and endometrium during human implantation.
Research Objective This research will define the dynamic mechanisms of human embryo implantation by integrating live imaging, spatial multi-omics, and gene editing in physiologically relevant stem cell-based models. Impact This…
Identifying and Overcoming Roadblocks to Hearing Restoration Using Human Induced Pluripotent Stem Cells
Research Objective To utilize human iPSCs as an in vitro model of hearing loss and overcome obstacles to hair cell regeneration by prompting post-mitotic supporting cells to re-enter the cell…
Mechanisms underlying dosage sensitivity in developmental disorders
Research Objective This proposal leverages pluripotent stem cell models to understand the principles by which changes in gene and protein dosage affect human neural and facial progenitor development and cause…
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…
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…
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…
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…