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Cell-penetrating peptides as a delivery system for oligonucleotides

Drug Discovery World

Emerging as a new treatment option in rare and orphan disease areas, oligonucleotide therapeutics have matured into a drug class with a broad indication spectrum. Over the years, a range of different CPPs have been developed, including natural translocating proteins through to newly-designed computer-prediction sequences 3.

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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.

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