CliniCal Trials spoTlighT symposium has exciting applications for inherited diseases such as allele-specific silencing for autosomal-dominant mutants or activation of fetal genes as substitutes for mutant adult cognates. However, long-term transcriptional up-/down-regulation of target genes has many other applications since promoter/enhancer-specific small RNAs/oligos target non-coding sense/antisense RNAs including long ones, i.e. the very RNAs that appear to drive the complex digital regulation of human epigenetic processes and inherent pathologies. Therefore, our approach is discussed in terms of new therapeutic/prophylactic avenues for 1) epigenetic pathologies such as neurodevelopmental disorders originating from deleterious early life epigenetic programming (eg depression, schizophrenia), 2) therapeutic remodeling of imprinted gene expression (eg Angelman syndrome), 3) degenerative diseases and age-related disorders (eg Alzheimer's & Parkinson's diseases), 4) cancer and infectious diseases , 5) the ex vivo genesis of clinicalgrade iPS cells and 6) rising stem cell conversion.
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