The CuRe Trial: Cellular Therapy for In Utero Repair of Myelomeningocele
Therapeutic Candidate or Device Allogeneic Placenta-derived Mesenchymal Stem Cells Seeded on Cook Biodesign® Dural Graft Extracellular Matrix (PMSC-ECM) Indication Myelomeningocele (MMC) -or Spina Bifida -diagnosed prenatally Therapeutic Mechanism Placenta-derived mesenchymal…
Pre-Clinical To Clinical Gene Therapy Development For CMT4J
Therapeutic Candidate or Device AAV9 Gene Therapy For An Ultra-Rare Disease Called CMT4J Indication CMT4J or the FIG4 Gene Therapeutic Mechanism Gene Therapy through an Intrathecal One-Time Injection Unmet Medical…
MicroRNAs in Human Stem Cell Differentiation and Mental Disorders
Many mental disorders are closely associated with problems that occur during brain development in early life. For instance, by 2 years of age, autistic children have larger brains than normal…
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…
