OLIGONUCLEOTIDE COMPOSITIONS AND METHODS OF USE THEREOF

Among other things, the present disclosure provides C9orf72 oligonucleotides, compositions, and methods thereof. In some embodiments, the present disclosure provides methods for treating C9orf72-associated conditions, disorders or diseases, such as amyotrophic lateral sclerosis and frontotemporal de...

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Bibliographische Detailangaben
Hauptverfasser: LIU, Yuanjing, IWAMOTO, Naoki, ZHONG, Zhong, VARGEESE, Chandra, ANDREUCCI, Amy Jada
Format: Patent
Sprache:eng ; fre ; ger
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Zusammenfassung:Among other things, the present disclosure provides C9orf72 oligonucleotides, compositions, and methods thereof. In some embodiments, the present disclosure provides methods for treating C9orf72-associated conditions, disorders or diseases, such as amyotrophic lateral sclerosis and frontotemporal dementia.