Article

Diplotype-based expanded use of individualized allele-specific antisense oligonucleotides

2025-09-17

Abstract excerpt

<title>Abstract</title> <p>While an accelerated FDA process enables development and treatment trials of ASOs for individual patients within a year, it does not scale to patient communities. We evaluated a diplotype-based pathway for broad use of individualized ASOs whose pre-mRNA targets contain common single nucleotide variants. Two such ASOs that had shown safety and effectiveness in individual patients with SC...

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Literature Corpus work: 09672f43-e494-5460-8387-634b37d0acb0
DOI: 10.21203/rs.3.rs-7603916/v1

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