Article

TALEN-induced contraction of CTG trinucleotide repeats in myotonic dystrophy type 1 cells

2023-10-18

Abstract excerpt

Trinucleotide repeat expansions are the cause of two dozen neurodegenerative and developmental disorders. One of these, myotonic dystrophy type 1 (Steinert disease, or DM1) is due to the expansion of a CTG triplet in the 3’ UTR of the DMPK gene. We used highly specific DNA endonucleases to induce a double-strand break in the repeat tract to contract it below pathological length. Expression of a TALE Nuclease (TAL...

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Literature Corpus work: 64efb79a-3b63-5d13-8217-c2e7002793b9
DOI: 10.1101/2023.10.14.562330

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