Precise Targeted Cleavage of a r(CUG) Repeat Expansion in Cells by Using a Small-Molecule–Deglycobleomycin Conjugate
Author:
Affiliation:
1. Department of Chemistry, The Scripps Research Institute, 130 Scripps Way, Jupiter, Florida 33458, United States
2. Department of Chemistry, The Scripps Research Institute, 10550 North Torrey Pines Road, La Jolla, California 92037, United States
Funder
National Cancer Institute
National Institute of Neurological Disorders and Stroke
Division of Graduate Education
Scripps Research Institute
Publisher
American Chemical Society (ACS)
Subject
Molecular Medicine,General Medicine,Biochemistry
Link
https://pubs.acs.org/doi/pdf/10.1021/acschembio.0c00036
Reference33 articles.
1. Methods to identify and optimize small molecules interacting with RNA (SMIRNAs)
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3. A novel gene containing a trinucleotide repeat that is expanded and unstable on Huntington's disease chromosomes
4. Molecular basis of myotonic dystrophy: Expansion of a trinucleotide (CTG) repeat at the 3′ end of a transcript encoding a protein kinase family member
5. Myotonic Dystrophy Type 2 Caused by a CCTG Expansion in Intron 1 of ZNF9
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