Systemic Delivery of a Peptide-Linked Morpholino Oligonucleotide Neutralizes Mutant RNA Toxicity in a Mouse Model of Myotonic Dystrophy-Reference-Cited by-同舟云学术

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

Published:2013-04 Issue:2 Volume:23 Page:109-117
ISSN:2159-3337
Container-title:Nucleic Acid Therapeutics
language:en
Short-container-title:Nucleic Acid Therapeutics

Author:

Leger Andrew J.¹,Mosquea Leocadia M.¹,Clayton Nicholas P.¹,Wu I-Huan¹,Weeden Timothy¹,Nelson Carol A.¹,Phillips Lucy¹,Roberts Errin¹,Piepenhagen Peter A.¹,Cheng Seng H.¹,Wentworth Bruce M.¹

Affiliation:

1. Genzyme, a Sanofi Company, Framingham, Massachusetts.

Publisher

Mary Ann Liebert Inc

Subject

Drug Discovery,Genetics,Molecular Biology,Molecular Medicine,Biochemistry

Link

http://www.liebertpub.com/doi/pdf/10.1089/nat.2012.0404

Reference33 articles.

1. Vectorization of morpholino oligomers by the (R-Ahx-R)4 peptide allows efficient splicing correction in the absence of endosomolytic agents

2. Rationally Designed Small Molecules Targeting the RNA That Causes Myotonic Dystrophy Type 1 Are Potently Bioactive

3. RNA pathogenesis of the myotonic dystrophies

4. Morpholino Oligomer–Mediated Exon Skipping Averts the Onset of Dystrophic Pathology in the mdx Mouse

5. Dynamic Combinatorial Selection of Molecules Capable of Inhibiting the (CUG) Repeat RNA−MBNL1 Interaction In Vitro: Discovery of Lead Compounds Targeting Myotonic Dystrophy (DM1)

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