Ribonucleic acid (RNA) is found in all living cells. As a transcription of DNA, RNA is the link to a functional proteom. Depending on its function, RNA occurs in different forms. Since diseases are often caused by a missing or dysfunctional protein, RNA-based therapeutics become more and more popular in the treatment of various diseases.
Traditional medicine, based on antibodies and small molecules, directly targets the disease-causing protein as soon as it is produced (translated). Consequently, the protein function is blocked.
However, this approach fails to treat diseases caused by reduced protein production and must be dosed carefully.
Of particular interest for drug development is cell process regulating RNA, such as small interfering RNA (siRNA). Complementary binding to protein-coding mRNA blocks the translation of specific proteins.
In case of siRNA malfunction, this cellular regulation is mimicked in RNA-based therapeutics by antisense oligonucleotides.
RNA-based therapeutics are changing the way diseases are treated and even enabling the treatment of previously untreatable diseases.
Advance research with us and synthesize modified RNA in your lab with Kilobaser modified RNA cartridge.
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