Peptide Nucleic Acid Promotes Systemic Dystrophin Expression and Functional Rescue in Dystrophin-deficient mdx Mice

Peptide Nucleic Acid Promotes Systemic Dystrophin Expression and Functional Rescue in Dystrophin-deficient mdx Mice

अन्य संस्करण दिखाएं (1)

Antisense oligonucleotide (AO)-mediated exon-skipping therapeutics shows great promise for Duchenne muscular dystrophy (DMD) patients. However, recent failure with drisapersen, an AO candidate drug in phase 3 trial, highlights the importance of exploring other effective AO chemistries for DMD. Previ...

पूर्ण विवरण

ग्रंथसूची विवरण
मुख्य लेखकों: Xianjun Gao, Xiaoyong Shen, Xue Dong, Ning Ran, Gang Han, Limin Cao, Ben Gu, HaiFang Yin
स्वरूप: लेख
भाषा:English
प्रकाशित: Elsevier 2015-01-01
श्रृंखला:Molecular Therapy: Nucleic Acids
विषय:
antisense oligonucleotide
Duchenne muscular dystrophy
dystrophin
exon skipping
peptide nucleic acid
ऑनलाइन पहुंच:http://www.sciencedirect.com/science/article/pii/S2162253116300403

इंटरनेट

http://www.sciencedirect.com/science/article/pii/S2162253116300403

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