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RNA Therapeutics: A Novel Approach to Treating Diseases

Roots Analysis

Coding RNAs include messenger RNA (mRNA) and short interfering RNA (siRNA), which encode proteins and silence gene expression, respectively. ASOs are short single-stranded nucleotides that bind to specific messenger RNAs and prevent the production of a particular protein.

RNA 40
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The use of base editing in stem-cell based therapies

Drug Discovery World

Adult fibroblasts were cultured in a cocktail of transcription factors to reactivate embryonic gene expression patterns 2. For editing of stem cells, this raises the possibility of designing iPSC-derived cell therapies where gene regulatory networks can be altered through the silencing or activation of specific genes and pathways.