Review Article on Nucleic Acid Therapeutics - Full Text | Biotech Message Board Posts


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Msg  39392 of 39910  at  9/26/2021 2:27:05 PM  by

Fall1980


Review Article on Nucleic Acid Therapeutics - Full Text

Includes info on specific drugs, cost of treatment. From Nature Nanotechnology, June 2021. H/T to OP Bigbiotechbull on another mb.
 
The current landscape of nucleic acid therapeutics 
 
[Abstract] The increasing number of approved nucleic acid therapeutics demonstrates the potential to treat diseases by targeting their genetic blueprints in vivo. Conventional treatments generally induce therapeutic effects that are transient because they target proteins rather than underlying causes. In contrast, nucleic acid therapeutics can achieve long-lasting or even curative effects via gene inhibition, addition, replacement or editing. Their clinical translation, however, depends on delivery technologies that improve stability, facilitate internalization and increase target affinity. Here, we review four platform technologies that have enabled the clinical translation of nucleic acid therapeutics: antisense oligonucleotides, ligand-modified small interfering RNA conjugates, lipid nanoparticles and adeno-associated virus vectors. For each platform, we discuss the current state-of-the-art clinical approaches, explain the rationale behind its development, highlight technological aspects that facilitated clinical translation and provide an example of a clinically relevant genetic drug. In addition, we discuss how these technologies enable the development of cutting-edge genetic drugs, such as tissue-specific nucleic acid bioconjugates, messenger RNA and gene-editing therapeutics.
 


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