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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. In the last few years, researchers have become interested in using in vitro transcribed (IVT) mRNA as a drug delivery agent.

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. Base editing uses a guide RNA in partnership with a nickase version of Cas9, mutated to cut only a single strand of DNA, along with a deaminase enzyme to enable alteration of a single nucleotide 23,24.