Disease Focus: Developmental Disorders


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 closure of the developing spinal cord, resulting in neurological damage to the exposed cord.  This damage leads to lifelong lower body paralysis, and bladder and […]

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 […]

Functional genomics to study cellular convergence across ASD risk genes in neurodevelopment

Research Objective Our objective is to enable scalable genetic screening to study how neurogenesis is impacted by risk genes implicated in human psychiatric disorders. Impact We will develop and apply state-of-the-art genomic analysis to seek mechanisms and disease modifying solutions. Major Proposed Activities Identifying and validating efficient gRNA for gene editing Identify risk gene effects […]

2016 Gordon Research Conference on Craniofacial Morphogenesis and Tissue Regeneration

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 stem cells (PMSCs) act by a paracrine mechanism, secreting a variety of growth factors, cytokines, and extracellular vesicles. This secretory profile is unique to PMSCs […]

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 Need CMT4J is an ultra-rare disorder that presently lacks any available treatment options and represents an underserved orphan population. Project Objective Successful Filing of an […]

Stem Cells Craniofacial Development and Disease

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 kids, likely due to, at least in part, excess production of neurons and support cells, the building blocks of the nervous system. In autistic brains, […]

Preeclampsia and Stem Cell Research: Mana Parast, UC-San Diego

Preeclampsia is a disorder of the placenta that complicates 5-8% of all pregnancies worldwide and is the leading cause of maternal death in the developed world. It’s also the leading cause of fetal growth restriction and there’s no cure except to deliver the baby. Due to this fact preeclampsia is the number one cause of […]

Preeclampsia and Stem Cell Research: The Parent’s Perspective

Preeclampsia is a disorder of the placenta that complicates 5-8% of all pregnancies worldwide and is the leading cause of maternal death in the developed world. It’s also the leading cause of fetal growth restriction and there’s no cure except to deliver the baby. Due to this fact preeclampsia is the number one cause of […]

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