Immortalized Muscle Cell Model to Test the Exon Skipping Efficacy for Duchenne Muscular Dystrophy

Immortalized Muscle Cell Model to Test the Exon Skipping Efficacy for Duchenne Muscular Dystrophy

Duchenne muscular dystrophy (DMD) is a lethal genetic disorder that most commonly results from mutations disrupting the reading frame of the dystrophin (DMD) gene. Among the therapeutic approaches employed, exon skipping using antisense oligonucleotides (AOs) is one of the most promising strategies....

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Bibliographic Details
Main Authors: Quynh Nguyen, Toshifumi Yokota
Format: Article
Language:English
Published: MDPI AG 2017-10-01
Series:Journal of Personalized Medicine
Subjects:
Duchenne/Becker muscular dystrophy (DMD/BMD)
antisense oligonucleotide-mediated exon skipping therapy
hDMD mice
human telomerase reverse transcriptase (hTERT)
cyclin-dependent-kinase 4 (Cdk4)
phosphorodiamidate morpholino oligomers (PMOs or morpholinos)
C2C12
dystrophin-glycoprotein complex (DGC)
golodirsen (SRP-4053)
NS-065/NCNP-01
Online Access:https://www.mdpi.com/2075-4426/7/4/13

Internet

https://www.mdpi.com/2075-4426/7/4/13

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