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 pharmacok...

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Bibliografski detalji
Izdano u:Hum Mol Genet
Glavni autori: Zhao, Xin, Feng, Zhihua, Ling, Karen K. Y., Mollin, Anna, Sheedy, Josephine, Yeh, Shirley, Petruska, Janet, Narasimhan, Jana, Dakka, Amal, Welch, Ellen M., Karp, Gary, Chen, Karen S., Metzger, Friedrich, Ratni, Hasane, Lotti, Francesco, Tisdale, Sarah, Naryshkin, Nikolai A., Pellizzoni, Livio, Paushkin, Sergey, Ko, Chien-Ping, Weetall, Marla
Format: Artigo
Jezik:Inglês
Izdano: Oxford University Press 2016
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Articles
Online pristup:https://ncbi.nlm.nih.gov/pmc/articles/PMC5062580/
https://ncbi.nlm.nih.gov/pubmed/26931466
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/hmg/ddw062
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