Article

EDSpliCE, a CRISPR-Cas9 gene editing platform to rescue splicing, effectively corrects inherited retinal dystrophy-associated splicing defects

2024-03-30

Abstract excerpt

<h4>Background</h4> Correct splicing of transcripts is essential to ensure the production of functional gene products in eukaryotic cells. Missplicing of transcripts has been identified as the underlying molecular mechanisms behind various disease-causing variants in a wide range of inherited genetic conditions. Achieving therapeutic splicing correction is possible through antisense oligonucleotide and CRISPR/Cas...

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Literature Corpus work: 3a8d6bff-84c4-5fad-a84d-002f1d57430a
DOI: 10.1101/2024.03.27.587013

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