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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. Oligonucleotides act on the RNA level through different molecular pathways, but there is a major drawback when using them as drugs: their poor bioavailability and cellular uptake.

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RNA Therapeutics: A Novel Approach to Treating Diseases

Roots Analysis

RNA therapeutics are a novel class of biopharmaceuticals that harness the power of RNA molecules for the treatment and prevention of a wide range of disorders, including oncological, and genetic disorders as well as infectious diseases. Non-coding RNAs include antisense oligonucleotides (ASOs) and RNA aptamers.

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