Therapeutic impact of systemic AAV-mediated RNA interference in a mouse model of myotonic dystrophy.
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PABPN1 gene therapy for oculopharyngeal muscular dystrophySystemic delivery of adeno-associated viral vectors.Special Issue: Gene Therapy with Emphasis on RNA InterferenceCRISPR/Cas9-Mediated Deletion of CTG Expansions Recovers Normal Phenotype in Myogenic Cells Derived from Myotonic Dystrophy 1 Patients.Pre-clinical Safety and Off-Target Studies to Support Translation of AAV-Mediated RNAi Therapy for FSHD.Daunorubicin reduces MBNL1 sequestration caused by CUG-repeat expansion and rescues cardiac dysfunctions in a Drosophila model of myotonic dystrophy.
P2860
Therapeutic impact of systemic AAV-mediated RNA interference in a mouse model of myotonic dystrophy.
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2015 nî lūn-bûn
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2015年の論文
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2015年論文
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2015年論文
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name
Therapeutic impact of systemic ...... e model of myotonic dystrophy.
@ast
Therapeutic impact of systemic ...... e model of myotonic dystrophy.
@en
type
label
Therapeutic impact of systemic ...... e model of myotonic dystrophy.
@ast
Therapeutic impact of systemic ...... e model of myotonic dystrophy.
@en
prefLabel
Therapeutic impact of systemic ...... e model of myotonic dystrophy.
@ast
Therapeutic impact of systemic ...... e model of myotonic dystrophy.
@en
P2093
P2860
P356
P1476
Therapeutic impact of systemic ...... se model of myotonic dystrophy
@en
P2093
Darren R Bisset
Gregory T Carter
Jessica Wei
Joel R Chamberlain
Maja Zavaljevski
Michael D Weiss
P2860
P304
P356
10.1093/HMG/DDV219
P577
2015-06-16T00:00:00Z