OLIGONUCLEOTIDES USEFUL FOR MODULATION OF SPLICING

Described herein are oligonucleotides (e.g., single-stranded oligonucleotides) and compositions thereof for targeting a mutation in the spliceosome, such as the U 1 small nuclear RNA (snRNA), as well as related methods of use.

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Bibliographic Details
Main Authors Agrawal, Anant A, Seiler, Michael W, Smith, Peter, Smith, Jesse Jerome, Brown, Duncan
Format Patent
LanguageEnglish
Published 18.01.2024
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Summary:Described herein are oligonucleotides (e.g., single-stranded oligonucleotides) and compositions thereof for targeting a mutation in the spliceosome, such as the U 1 small nuclear RNA (snRNA), as well as related methods of use.
Bibliography:Application Number: US202118033748