Wednesday, January 30, 2013

Human Embryonic Stem Cells as the Model System for Understanding Molecular Controls in Human Central Nervous System (CNS) Development


San Diego Regenerative Medicine Institute and Xcelthera announce the publication of Dr. Parsons’ original research article supported by the National Institutes of Health, titled “Genome-Scale Mapping of MicroRNA Signatures in Human Embryonic Stem Cell Neurogenesis”, in Molecular Medicine & Therapeutics at http://dx.doi.org/10.4172/2324-8769.100010 & http://www.scitechnol.com/2324-8769/2324-8769-1-105.php .

Understanding the much more complex human embryonic development has been hindered by the restriction on human embryonic and fetal materials as well as the limited availability of human cell types and tissues for study. In particular, there is a fundamental gap in our knowledge regarding the molecular networks and pathways underlying the CNS and heart formation in human embryonic development. The enormous diversity of human somatic cell types and the highest order of complexity of human genomes, cells, tissues, and organs among all the eukaryotes pose a big challenge for characterizing, identifying, and validating functional elements in human embryonic development in a comprehensive manner. Derivation of human embryonic stem cells (hESCs) provides a powerful in vitro model system to investigate the molecular controls in human embryonic development as well as an unlimited source to generate the diversity of human cell types and subtypes across the spectrum of development stages for repair. Development and utilization of hESC models of human embryonic development will facilitate rapid progress in identification of molecular and genetic therapeutic targets for the prevention and treatment of human diseases. To tackle the shortcomings in conventional approaches, previously, we found that pluripotent hESCs maintained under the defined culture conditions can be uniformly converted into a specific lineage by small molecule induction. Our innovative small molecule direct induction approach renders a cascade of neuronal or cardiac lineage-specific progression directly from the pluripotent state of hESCs, providing much-needed in vitro model systems for investigating the human CNS development and heart formation in embryogenesis. This technology breakthrough not only opens the door for further identification of the developmental networks in human embryonic neurogenesis and cardiogenesis in a comprehensive manner, but also offers means for small-molecule-mediated direct control and modulation of the pluripotent fate of hESCs when deriving an unlimited supply of clinically-relevant lineages for regenerative medicine. Recent advances in large-scale profiling of developmental regulators in high-resolution provide powerful genome-wide high-throughput approaches that will lead to great advances in our understanding of the global phenomena of human embryogenesis. Profiling novel hESC models of human embryonic neurogenesis and cardiogenesis using genome-wide approaches, including employing ChIP-on-chip and microRNA mapping, will reveal molecular controls and the underlying mechanisms in hESC neuronal and cardiomyocyte fate decisions. Unveiling developmental networks during human embryonic neurogenesis and cardiogenesis using novel hESC models will contribute tremendously to our knowledge regarding molecular embryogenesis in human development, thereby, reveal potential therapeutic targets and aid the development of more optimal stem-cell-mediated therapeutic strategies for the prevention and treatment of CNS and heart diseases. The outcome of such research will potentially shift current research to create new scientific paradigms for developmental biology and stem cell research.

MicroRNAs are small, evolutionarily conserved non-coding RNAs that modulate gene expression by inhibiting mRNA translation and promoting mRNA degradation. MicroRNAs act as the governors of gene expression networks, thereby modify complex cellular phenotypes in development or disorders. MicroRNA expression profiling using microarrays is a powerful high-throughput tool capable of monitoring the regulatory networks of the entire genome and identifying functional elements in development in high resolution. To uncover key regulators in human CNS development during embryogenesis, in this Dr. Parsons’ research article, genome-scale profiling of microRNA differential expression patterns during hESC neuronal lineage-specific progression was used to identify novel sets of stage-specific human embryonic neurogenic microRNAs. We found that the prominent pluripotence-associated microRNAs were silenced and microRNAs involved in developmental networks were drastically up-regulated. Our findings suggest that these hESC neuronal derivatives have acquired a neuronal lineage-specific identity by silencing pluripotence-associated miRNAs and inducing the expression of miRNAs linked to regulating human CNS development to high levels, therefore, highly neurogenic in vitro and in vivo. Our study provides critical insight into molecular neurogenesis in human embryonic development as well as offers an adequate human neurogenic cell source in high purity and large quantity for CNS tissue engineering and developing safe and effective stem cell therapy to restore the lost nerve tissue and function. 

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