Systemic Delivery of a Peptide-Linked Morpholino Oligonucleotide Neutralizes Mutant RNA Toxicity in a Mouse Model of Myotonic Dystrophy

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Keyword(s):  
Mouse Model ◽  
Myotonic Dystrophy ◽  
Morpholino Oligonucleotide ◽  
Systemic Delivery ◽  
Rna Toxicity

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Mahua Mandal ◽  
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Keyword(s):  
Mouse Model ◽  
Myotonic Dystrophy ◽  
Age Of Onset ◽  
Rna Toxicity

O.7 Systemic delivery of RNase H-active antisense oligonucleotides reverses RNA dominance in a mouse model of myotonic dystrophy

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Vol 21 (9-10) ◽  
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Author(s):  
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A.J. Leger ◽  
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A.R. MacLeod ◽  
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Keyword(s):  
Mouse Model ◽  
Myotonic Dystrophy ◽  
Antisense Oligonucleotides ◽  
Rnase H ◽  
Systemic Delivery

scholarly journals Systemic delivery of AAVrh74.tMCK.hCAPN3 rescues the phenotype in a mouse model for LGMD2A/R1

2021 ◽  
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Burcak Ozes ◽  
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Morgan Myers ◽  
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Keyword(s):  
Mouse Model ◽  
Systemic Delivery
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