Gene suppressing therapy for Pelizaeus-Merzbacher disease using artificial microRNA

Copy number increase or decrease of certain dosage-sensitive genes may cause genetic diseases with distinct phenotypes, conceptually termed genomic disorders. The most common cause of Pelizaeus-Merzbacher disease (PMD), an X-linked hypomyelinating leukodystrophy, is genomic duplication encompassing...

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Dades bibliogràfiques
Publicat a:JCI Insight
Autors principals: Li, Heng, Okada, Hironori, Suzuki, Sadafumi, Sakai, Kazuhisa, Izumi, Hitomi, Matsushima, Yukiko, Ichinohe, Noritaka, Goto, Yu-ichi, Okada, Takashi, Inoue, Ken
Format: Artigo
Idioma:Inglês
Publicat: American Society for Clinical Investigation 2019
Matèries:
Research Article
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC6542614/
https://ncbi.nlm.nih.gov/pubmed/31092737
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1172/jci.insight.125052
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