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Showing 1–2 of 2 results
Advanced filters: Author: "Rajeev Sivasankaran" Clear advanced filters

  • Huntington’s disease (HD) results from the abnormal expansion of CAG repeats in exon 1 of the HTT gene. Here, the authors show that orally available, brain penetrant molecule branaplam lowers HTT transcript by promoting inclusion of a poison exon or pseudoexon.

    • Caroline Gubser Keller
    • Youngah Shin
    • Rajeev Sivasankaran
    ResearchOpen Access
    Nature Communications
    Volume: 13, P: 1-11

  • A high-throughput screen identified a small molecule that promoted inclusion of SMN2 exon 7, increased SMN2 protein levels and extended survival in a SMA mouse model through stabilization of the interaction between SMN2 pre-mRNA and U1 snRNP complex.

    • James Palacino
    • Susanne E Swalley
    • Rajeev Sivasankaran
    Research
    Nature Chemical Biology
    Volume: 11, P: 511-517