Mechanistic studies of a small-molecule modulator of SMN2 splicing
نویسندگان
چکیده
منابع مشابه
Pharmacokinetics, pharmacodynamics, and efficacy of a small-molecule SMN2 splicing modifier in mouse models of spinal muscular atrophy
Spinal muscular atrophy (SMA) is caused by the loss or mutation of both copies of the survival motor neuron 1 (SMN1) gene. The related SMN2 gene is retained, but due to alternative splicing of exon 7, produces insufficient levels of the SMN protein. Here, we systematically characterize the pharmacokinetic and pharmacodynamics properties of the SMN splicing modifier SMN-C1. SMN-C1 is a low-molec...
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ژورنال
عنوان ژورنال: Proceedings of the National Academy of Sciences
سال: 2018
ISSN: 0027-8424,1091-6490
DOI: 10.1073/pnas.1800260115