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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Publicat a:Hum Mol Genet
Autors principals: 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
Idioma:Inglês
Publicat: Oxford University Press 2016
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Articles
Accés en línia: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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