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Showing 1–10 of 10 results
Advanced filters: Author: "Anastasia Khvorova" Clear advanced filters

  • Since the groundbreaking discovery of RNAi more than 25 years ago, several small interfering RNA (siRNA)-based therapies that target the liver have gained approval. This Review discusses principal considerations in siRNA-based drug development, focusing on the medicinal chemistry of siRNA design, the application of informatics, delivery platforms and future directions.

    • Qi Tang
    • Anastasia Khvorova
    Reviews
    Nature Reviews Drug Discovery
    P: 1-24

  • Therapeutic modulation of Janus kinase family enzymes is an established approach for inflammatory and autoimmune skin diseases. Here the authors rationally design small interfering RNAs to enable single Janus kinase targeting and test this new therapeutic approach in a skin disease model for maintaining efficacy and improving selectivity.

    • Qi Tang
    • Hassan H. Fakih
    • John E. Harris
    ResearchOpen Access
    Nature Communications
    Volume: 14, P: 1-15

  • Resistance of gRNA to ubiquitous ribonucleases is required for CRISPR-Cas9-based therapeutics. Here, the authors explore chemical modifications at all positions of the crRNA guide and tracrRNA cofactor, and identify modified versions that are more potent and stable than their unmodified counterparts in editing human cells.

    • Aamir Mir
    • Julia F. Alterman
    • Erik J. Sontheimer
    ResearchOpen Access
    Nature Communications
    Volume: 9, P: 1-9

  • An additional layer of RNA regulation in which RNAs encoded by genes and pseudogenes compete for microRNAs could offer new opportunities for oligonucleotide therapeutics.

    • Anastasia Khvorova
    • Alexey Wolfson
    News & Views
    Nature Biotechnology
    Volume: 30, P: 58-59

  • Delivery of siRNA into cells is achieved by neutralizing the negative charge of the phosphate backbone in a reversible manner.

    • Anastasia Khvorova
    • Maire F Osborn
    • Matthew R Hassler
    News & Views
    Nature Biotechnology
    Volume: 32, P: 1197-1198

  • Refinements in the chemistries employed in oligonucleotide therapeutics have galvanized clinical progress. The complex interplay between chemical modifications and integration into sequence architecture is discussed in the context of antisense and small-interfering RNA drugs.

    • Anastasia Khvorova
    • Jonathan K Watts
    Reviews
    Nature Biotechnology
    Volume: 35, P: 238-248