A Guide to RNA Therapeutics
An in-depth guide to the evolving world of RNA therapeutics.
RNA Therapeutics Guide
RNA-based therapies are reshaping the future of medicine, from mRNA vaccines to gene-silencing drugs. This eBook offers a practical overview of the RNA therapeutic landscape, focusing on the science, strategies and challenges behind developing effective RNA treatments.
What You'll Learn
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The therapeutic mechanisms behind RNA types, including mRNA, siRNA, ASOs, aptamers, and circRNA
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Structural elements of therapeutic mRNA, and how 5' caps, UTRs, codon optimization, and poly(A) tails affect stability and translation
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Best practices for mRNA production, using integrated gene synthesis and in vitro transcription workflows
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Advanced QC strategies, including full-length RNA sequencing to assess transcript integrity, poly(A) tail length, and product purity
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Clinical and emerging applications, from FDA-approved drugs to cancer vaccines and treatments for rare neurological diseases