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Showing 1–2 of 2 results
Advanced filters: Author: Vijayalakshmi Gabbeta Clear advanced filters

  • Here the authors describe the discovery of a class of small molecule splicing modifiers which are orally bioavailable, cross the blood-brain barrier, and lower levels of huntingtin in a mouse model of Huntington’s disease (HD).

    • Anuradha Bhattacharyya
    • Christopher R. Trotta
    • Stuart W. Peltz
    ResearchOpen Access
    Nature Communications
    Volume: 12, P: 1-12

  • Drugs that modify RNA splicing are promising treatments for many genetic diseases. Here the authors show that deep learning strategies can predict drug targets, strongly supporting the use of in silico approaches to expand the therapeutic potential of drugs that modulate RNA splicing.

    • Dadi Gao
    • Elisabetta Morini
    • Susan A. Slaugenhaupt
    ResearchOpen Access
    Nature Communications
    Volume: 12, P: 1-15