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Gene Therapy for Muscular Dystrophy: Lessons Learned and Path Forward

Our Translational Gene Therapy Center has used small molecules for exon skipping and mutation suppression and gene transfer to replace or provide surrogate genes as tools for molecular-based approaches for the treatment of muscular dystrophies. Exon skipping is targeted at the pre-mRNA level allowin...

詳細記述

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書誌詳細
主要な著者: Mendell, Jerry R., Rodino-Klapac, Louise, Sahenk, Zarife, Malik, Vinod, Kaspar, Brian K., Walker, Christopher M., Clark, K. Reed
フォーマット: Artigo
言語:Inglês
出版事項: 2012
主題:
オンライン・アクセス:https://ncbi.nlm.nih.gov/pmc/articles/PMC3492936/
https://ncbi.nlm.nih.gov/pubmed/22609847
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.neulet.2012.04.078
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