A Selective Alkylating Agent for CTG Repeats in Myotonic Dystrophy Type 1

Autor:	JuYeon Lee, Ke Li, Steven C. Zimmerman
Rok vydání:	2022
Předmět:	Alkylating Agents Humans Myotonic Dystrophy Molecular Medicine DNA General Medicine Ligands Trinucleotide Repeat Expansion Biochemistry
Zdroj:	ACS Chemical Biology. 17:1103-1110
ISSN:	1554-8937 1554-8929
Popis:	Disease intervention at the DNA level generally has been avoided because of off-target effects. Recent advances in genome editing technologies using CRISPR-Cas9 have opened a new era in DNA-targeted therapeutic approaches. However, delivery of such systems remains a major challenge. Here, we report a selective DNA-modifying small molecule that targets a disease-specific structure and mismatches involved in myotonic dystrophy type 1 (DM1). This ligand alkylates T-T mismatch-containing hairpins formed in the expanded CTG repeats (d(CTG)
Databáze:	OpenAIRE
Externí odkaz:	https://explore.openaire.eu/search/publication?articleId=doi_dedup___::8878b2b6c34af84a69233d9e4fa04cfb https://doi.org/10.1021/acschembio.1c00949 Zobrazit plný text záznamu