scholarly journals HTS-Compatible Patient-Derived Cell-Based Assay to Identify Small Molecule Modulators of Aberrant Splicing in Myotonic Dystrophy Type 1

2010 ◽  
Vol 4 ◽  
pp. 9-18 ◽  
Author(s):  
Debra A. O’Leary ◽  
Leonardo Vargas ◽  
Orzala Sharif ◽  
Michael E. Garcia ◽  
Yury J. Sigal ◽  
...  
Keyword(s):  
Myotonic Dystrophy ◽  
Small Molecule ◽  
Myotonic Dystrophy Type 1 ◽  
Myotonic Dystrophy Type ◽  
Aberrant Splicing ◽  
Small Molecule Modulators

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 Resveratrol corrects aberrant splicing of RYR1 pre-mRNA and Ca2+ signal in myotonic dystrophy type 1 myotubes

2020 ◽  
Vol 15 (9) ◽  
pp. 1757
Author(s):  
Massimo Santoro ◽  
Roberto Piacentini ◽  
Alessia Perna ◽  
Eugenia Pisano ◽  
Anna Severino ◽  
...  
Keyword(s):  
Myotonic Dystrophy ◽  
Myotonic Dystrophy Type 1 ◽  
Myotonic Dystrophy Type ◽  
Aberrant Splicing

scholarly journals Small molecule kinase inhibitors alleviate different molecular features of myotonic dystrophy type 1

RNA Biology ◽  
2014 ◽  
Vol 11 (6) ◽  
pp. 742-754 ◽  
Author(s):  
Marzena Wojciechowska ◽  
Katarzyna Taylor ◽  
Krzysztof Sobczak ◽  
Marek Napierala ◽  
Wlodzimierz J Krzyzosiak
Keyword(s):  
Myotonic Dystrophy ◽  
Small Molecule ◽  
Kinase Inhibitors ◽  
Myotonic Dystrophy Type 1 ◽  
Myotonic Dystrophy Type ◽  
Molecular Features

scholarly journals Manumycin A corrects aberrant splicing of Clcn1 in myotonic dystrophy type 1 (DM1) mice

2013 ◽  
Vol 3 (1) ◽  
Author(s):  
Kosuke Oana ◽  
Yoko Oma ◽  
Satoshi Suo ◽  
Masanori P. Takahashi ◽  
Ichizo Nishino ◽  
...  
Keyword(s):  
Myotonic Dystrophy ◽  
Myotonic Dystrophy Type 1 ◽  
Myotonic Dystrophy Type ◽  
Aberrant Splicing

scholarly journals Molecular mechanisms responsible for aberrant splicing of SERCA1 in myotonic dystrophy type 1

2007 ◽  
Vol 16 (23) ◽  
pp. 2834-2843 ◽  
Author(s):  
S.-i. Hino ◽  
S. Kondo ◽  
H. Sekiya ◽  
A. Saito ◽  
S. Kanemoto ◽  
...  
Keyword(s):  
Myotonic Dystrophy ◽  
Molecular Mechanisms ◽  
Myotonic Dystrophy Type 1 ◽  
Myotonic Dystrophy Type ◽  
Aberrant Splicing

SEVERE CONGENITAL MYOTONIC DYSTROPHY TYPE 1

2018 ◽  
Vol 97 (1) ◽  
pp. 78-81
Author(s):  
E.A. Mamaeva ◽  
◽  
L.A. Fedorova ◽  
S.E. Voronovich ◽  
V.D. Nazarov ◽  
...  
Keyword(s):  
Myotonic Dystrophy ◽  
Myotonic Dystrophy Type 1 ◽  
Myotonic Dystrophy Type ◽  
Congenital Myotonic Dystrophy
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