Oligonucleotide compositions and methods of use thereof
AssigneeWave Life Sciences Ltd.
InventorsYuanjing Liu, Naoki Iwamoto, Chandra Vargeese, Zhong Zhong, Amy Jada Andreucci, Susovan Mohapatra
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Abstract
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.
Claims30
An oligonucleotide comprising at least one modification of a sugar, or base, wherein the base sequence of the oligonucleotide is or comprises at least 15 contiguous bases of a base sequence that is identical with or complementary to a base sequence of a C9orf72 gene or a transcript thereof,
- wherein oligonucleotides of the plurality comprise at least one non-negatively charged internucleotidic linkage comprising a guanidine moiety.
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Dependencies are inferred from the claim text.