Triplex DNA: The next platform for oligonucleotide drug development

    Project Details

    Description

    AMS Springboard Award (£125K) to develop modified triplex-forming oligonucleotides for gene-targeting in living cells (due to start July). The project stems from a recent single author paper (NAR 2021) that addressed a long-standing problem with binding to genomic DNA using modified oligonucleotides and established a cost-effective method for enzymatically generating such oligonucleotides on a solid support. The project will therefore establish a novel drug development pipeline that combines both array-based assembly and screening technologies, with classical in vivo validation. Developing triplex recognition as an alternative to antisense/siRNA would significantly reduce oligonucleotide dose as there are just two copies of DNA per diploid cell. The grant provides salary for an 18-month post-doctoral researcher, equipment, and consumables, and is further supported by Firebird Biomolecular Sciences (Florida, US) who will provide bespoke oligonucleotides.
    StatusActive
    Effective start/end date1/07/2430/06/26

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