Oligonucleotide compositions and methods of use thereof

A technology of oligonucleotides and compositions, applied in biochemical equipment and methods, chemical instruments and methods, sugar derivatives, etc., can solve the problems of nucleic acid cell penetration and poor distribution
CN112004928APending Publication Date: 2020-11-27WAVE LIFE SCI LTD
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Patent Information

Authority / Receiving Office
CN · China
Current Assignee / Owner
WAVE LIFE SCI LTD
Publication Date
2020-11-27

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Abstract

Among other things, the present disclosure provides designed oligonucleotides, compositions, and methods of use thereof. In some embodiments, the present disclosure provides technologies useful for reducing levels of transcripts. In some embodiments, the present disclosure provides technologies useful for modulating transcript splicing. In some embodiments, provided technologies can alter splicingof a dystrophin (DMD) transcript. In some embodiments, the present disclosure provides methods for treating diseases, such as Duchenne muscular dystrophy, Becker's muscular dystrophy, etc.
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Description

[0001] Cross References to Related Applications

[0002] This application claims U.S. Provisional Application No. 62 / 656,949, filed April 12, 2018, U.S. Provisional Application No. 62 / 670,709, filed May 11, 2018, U.S. Provisional Application No., filed Aug. 07, 2018 Priority of 62 / 715,684, U.S. Provisional Application No. 62 / 723,375, filed August 27, 2018, and U.S. Provisional Application No. 62 / 776,432, filed December 06, 2018, each of which is incorporated by reference in its entirety into this article. Background technique

[0003] Oligonucleotides are useful in therapeutic, diagnostic, research, and nanomaterial applications. The therapeutic use of naturally occurring nucleic acids (e.g., unmodified DNA or RNA) may be limited, for example, because of the instability of naturally occurring nucleic acids to extracellular and intracellular nucleases and / or the Poor cell penetration and distribution. There is a need for new and improved oligonucleotides and oligonucleotide ...

Claims

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