Article

Rapid and scalable preclinical evaluation of personalized antisense oligonucleotide therapeutics using organoids derived from rare disease patients

2023-03-29

Abstract excerpt

Personalized antisense oligonucleotides (ASOs) have achieved positive results in the treatment of rare genetic disease. As clinical sequencing technologies continue to advance, the ability to identify rare disease patients harboring pathogenic genetic variants amenable to this therapeutic strategy will likely improve. Here, we describe a scalable platform for generating patient-derived cellular models and demonstr...

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Literature Corpus work: 4f1470fd-cc4f-5010-a502-fead407e4abc
DOI: 10.1101/2023.03.28.23287871

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