The CuRe Trial: Cellular Therapy for In Utero Myelomeningocele Repair and The CuRe Trial: Cellular Therapy for In Utero Repair of Myelomeningocele
UC Davis is conducting a clinical trial for in utero repair of myelomeningocele (MMC), the most severe form of spina bifida. MMC is a birth defect that occurs due to incomplete…
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…
High-Throughput Discovery of Embryo Formation Factors Using Stem Cell-Based Human Embryo Models
Research Objective To integrate emulsion microfluidics-based encapsulation with AI-powered imaging to develop a high-throughput pipeline for stem cell-based embryo models. Impact We will overcome key bottlenecks to drive breakthroughs in…
Unraveling the developmental path from altered hematopoietic stem cells to leukemia in Down syndrome
Research Objective Our goal is to map altered Trisomy 21 developmental hematopoiesis to understand the first steps of leukemia initiation in Myeloid Leukemia of Down syndrome Impact We will provide…
Hearing the Silence: Genome-wide Mapping of Cell-Type-Specific Silencers in the Developing Human Brain
Research Objective We will develop genome editing tools to identify silencers that regulate neural stem cell fate, uncovering key DNA elements that guide neurodevelopment and are disrupted in neurodevelopmental diseases…
A novel platform to rescue neurodevelopmental disorders caused by haploinsufficiency
Research Objective We develop a powerful platform to correct gene expression defects caused by haploinsufficiency and will show its ability to identify novel targets using iPSC-derived neurons and cortical organoids.…
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…
Mechanisms of Transcription Factor Haploinsufficiency in Human Congenital Heart Disease
Research Objective We aim to solve a 30 year old problem, which is to understand using human stem cells how certain genetic mutations cause human disease. Impact We currently don't…
Modeling of GATAD2B-associated neurodevelopmental disorder and NuRDopathies: Investigation of cellular & molecular anomalies altering neurodevelopment
Research Objective Human and animal models of NuRD-deficiency will identify NuRD-subtype function in context of neurogenesis. Multi-omic studies will identify/quantify molecular and cellular changes in NuRD-deficiency. Impact NuRD-deficiency causes several…