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    The chemical evolution of oligonucleotide therapies of clinical utility

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    Authors
    Khvorova, Anastasia
    Watts, Jonathan K.
    UMass Chan Affiliations
    Department of Biochemistry and Molecular Pharmacology
    Program in Molecular Medicine
    RNA Therapeutics Institute
    Document Type
    Journal Article
    Publication Date
    2017-03-01
    Keywords
    Biochemistry
    Biotechnology
    Chemicals and Drugs
    Medicinal Chemistry and Pharmaceutics
    Therapeutics
    
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    Link to Full Text
    https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5517098/
    Abstract
    After nearly 40 years of development, oligonucleotide therapeutics are nearing meaningful clinical productivity. One of the key advantages of oligonucleotide drugs is that their delivery and potency are derived primarily from the chemical structure of the oligonucleotide whereas their target is defined by the base sequence. Thus, as oligonucleotides with a particular chemical design show appropriate distribution and safety profiles for clinical gene silencing in a particular tissue, this will open the door to the rapid development of additional drugs targeting other disease-associated genes in the same tissue. To achieve clinical productivity, the chemical architecture of the oligonucleotide needs to be optimized with a combination of sugar, backbone, nucleobase, and 3'- and 5'-terminal modifications. A portfolio of chemistries can be used to confer drug-like properties onto the oligonucleotide as a whole, with minor chemical changes often translating into major improvements in clinical efficacy. One outstanding challenge in oligonucleotide chemical development is the optimization of chemical architectures to ensure long-term safety. There are multiple designs that enable effective targeting of the liver, but a second challenge is to develop architectures that enable robust clinical efficacy in additional tissues.
    Source
    Nat Biotechnol. 2017 Mar;35(3):238-248. doi: 10.1038/nbt.3765. Epub 2017 Feb 27. Link to article on publisher's site
    DOI
    10.1038/nbt.3765
    Permanent Link to this Item
    http://hdl.handle.net/20.500.14038/40328
    PubMed ID
    28244990
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    Link to Article in PubMed

    ae974a485f413a2113503eed53cd6c53
    10.1038/nbt.3765
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