scholarly journals Induction and reversal of myotonic dystrophy type 1 pre-mRNA splicing defects by small molecules

2013 ◽  
Vol 4 (1) ◽  
Author(s):  
Jessica L. Childs-Disney ◽  
Ewa Stepniak-Konieczna ◽  
Tuan Tran ◽  
Ilyas Yildirim ◽  
HaJeung Park ◽  
...  
Keyword(s):  
Small Molecules ◽  
Myotonic Dystrophy ◽  
Mrna Splicing ◽  
Myotonic Dystrophy Type 1 ◽  
Myotonic Dystrophy Type ◽  
Splicing Defects

scholarly journals Mitigating RNA Toxicity in Myotonic Dystrophy using Small Molecules

2019 ◽  
Vol 20 (16) ◽  
pp. 4017 ◽  
Author(s):  
Kaalak Reddy ◽  
Jana R. Jenquin ◽  
John D. Cleary ◽  
J. Andrew Berglund
Keyword(s):  
Small Molecules ◽  
Myotonic Dystrophy ◽  
Small Molecule ◽  
Disease Onset ◽  
Myotonic Dystrophy Type 1 ◽  
Myotonic Dystrophy Type ◽  
Rna Toxicity ◽  
Complex Mechanisms ◽  
Potential Use

This review, one in a series on myotonic dystrophy (DM), is focused on the development and potential use of small molecules as therapeutics for DM. The complex mechanisms and pathogenesis of DM are covered in the associated reviews. Here, we examine the various small molecule approaches taken to target the DNA, RNA, and proteins that contribute to disease onset and progression in myotonic dystrophy type 1 (DM1) and 2 (DM2).

scholarly journals Differences in splicing defects between the grey and white matter in myotonic dystrophy type 1 patients

PLoS ONE ◽  
2020 ◽  
Vol 15 (5) ◽  
pp. e0224912 ◽  
Author(s):  
Masamitsu Nishi ◽  
Takashi Kimura ◽  
Masataka Igeta ◽  
Mitsuru Furuta ◽  
Koichi Suenaga ◽  
...  
Keyword(s):  
White Matter ◽  
Myotonic Dystrophy ◽  
Myotonic Dystrophy Type 1 ◽  
Myotonic Dystrophy Type ◽  
Splicing Defects

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

2012 ◽  
Vol 7 (5) ◽  
pp. 856-862 ◽  
Author(s):  
Jessica L. Childs-Disney ◽  
Jason Hoskins ◽  
Suzanne G. Rzuczek ◽  
Charles A. Thornton ◽  
Matthew D. Disney
Keyword(s):  
Small Molecules ◽  
Myotonic Dystrophy ◽  
Myotonic Dystrophy Type 1 ◽  
Myotonic Dystrophy Type

Rationally Designed Small Molecules That Target Both the DNA and RNA Causing Myotonic Dystrophy Type 1

2015 ◽  
Vol 137 (44) ◽  
pp. 14180-14189 ◽  
Author(s):  
Lien Nguyen ◽  
Long M. Luu ◽  
Shaohong Peng ◽  
Julio F. Serrano ◽  
H. Y. Edwin Chan ◽  
...  
Keyword(s):  
Small Molecules ◽  
Myotonic Dystrophy ◽  
Myotonic Dystrophy Type 1 ◽  
Myotonic Dystrophy Type ◽  
Dna And Rna

scholarly journals Altered mRNA splicing of the skeletal muscle ryanodine receptor and sarcoplasmic/endoplasmic reticulum Ca2+-ATPase in myotonic dystrophy type 1

2005 ◽  
Vol 14 (15) ◽  
pp. 2189-2200 ◽  
Author(s):  
Takashi Kimura ◽  
Masayuki Nakamori ◽  
John D. Lueck ◽  
Pierre Pouliquin ◽  
Futoshi Aoike ◽  
...  
Keyword(s):  
Skeletal Muscle ◽  
Endoplasmic Reticulum ◽  
Myotonic Dystrophy ◽  
Ryanodine Receptor ◽  
Mrna Splicing ◽  
Myotonic Dystrophy Type 1 ◽  
Myotonic Dystrophy Type
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