Small Molecules that Target the Toxic RNA in Myotonic Dystrophy Type 2
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Title
Small Molecules that Target the Toxic RNA in Myotonic Dystrophy Type 2
Authors
Keywords
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Journal
ChemMedChem
Volume 9, Issue 11, Pages 2455-2462
Publisher
Wiley
Online
2014-06-18
DOI
10.1002/cmdc.201402095
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Related references
Note: Only part of the references are listed.- Targeting Toxic RNAs that Cause Myotonic Dystrophy Type 1 (DM1) with a Bisamidinium Inhibitor
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- Rationally Designed Small Molecules Targeting the RNA That Causes Myotonic Dystrophy Type 1 Are Potently Bioactive
- (2012) Jessica L. Childs-Disney et al. ACS Chemical Biology
- Antisense Oligonucleotides: Rising Stars in Eliminating RNA Toxicity in Myotonic Dystrophy
- (2012) Zhihua Gao et al. HUMAN GENE THERAPY
- Mouse model of muscleblind-like 1 overexpression: skeletal muscle effects and therapeutic promise
- (2012) Christopher M. Chamberlain et al. HUMAN MOLECULAR GENETICS
- From dynamic combinatorial ‘hit’ to lead: in vitro and in vivo activity of compounds targeting the pathogenic RNAs that cause myotonic dystrophy
- (2012) Leslie O. Ofori et al. NUCLEIC ACIDS RESEARCH
- Structure of the HIV-1 Frameshift Site RNA Bound to a Small Molecule Inhibitor of Viral Replication
- (2011) Ryan J. Marcheschi et al. ACS Chemical Biology
- Selective inhibition of MBNL1–CCUG interaction by small molecules toward potential therapeutic agents for myotonic dystrophy type 2 (DM2) †
- (2011) Chun-Ho Wong et al. NUCLEIC ACIDS RESEARCH
- Mutant (CCTG)n Expansion Causes Abnormal Expression of Zinc Finger Protein 9 (ZNF9) in Myotonic Dystrophy Type 2
- (2010) Olayinka Raheem et al. AMERICAN JOURNAL OF PATHOLOGY
- The Role of Flexibility in the Rational Design of Modularly Assembled Ligands Targeting the RNAs that Cause the Myotonic Dystrophies
- (2010) Matthew D. Disney et al. CHEMBIOCHEM
- Myotonic Dystrophies 1 and 2: Complex Diseases with Complex Mechanisms
- (2010) Benedikt Schoser et al. CURRENT GENOMICS
- The myotonic dystrophy type 2 (DM2) gene product zinc finger protein 9 (ZNF9) is associated with sarcomeres and normally localized in DM2 patients' muscles
- (2010) R. Massa et al. NEUROPATHOLOGY AND APPLIED NEUROBIOLOGY
- Rational and Modular Design of Potent Ligands Targeting the RNA That Causes Myotonic Dystrophy 2
- (2009) Melissa M. Lee et al. ACS Chemical Biology
- Expression of RNA CCUG Repeats Dysregulates Translation and Degradation of Proteins in Myotonic Dystrophy 2 Patients
- (2009) Elizabeth Salisbury et al. AMERICAN JOURNAL OF PATHOLOGY
- Reduction of the Rate of Protein Translation in Patients with Myotonic Dystrophy 2
- (2009) C. Huichalaf et al. JOURNAL OF NEUROSCIENCE
- Controlling the Specificity of Modularly Assembled Small Molecules for RNA via Ligand Module Spacing: Targeting the RNAs That Cause Myotonic Muscular Dystrophy
- (2009) Melissa M. Lee et al. JOURNAL OF THE AMERICAN CHEMICAL SOCIETY
- A simple ligand that selectively targets CUG trinucleotide repeats and inhibits MBNL protein binding
- (2009) J. F. Arambula et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- Pentamidine reverses the splicing defects associated with myotonic dystrophy
- (2009) M. B. Warf et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- Dynamic Combinatorial Selection of Molecules Capable of Inhibiting the (CUG) Repeat RNA−MBNL1 Interaction In Vitro: Discovery of Lead Compounds Targeting Myotonic Dystrophy (DM1)
- (2008) Peter C. Gareiss et al. JOURNAL OF THE AMERICAN CHEMICAL SOCIETY
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