Publication number: 20250066775
Abstract: Among other things, the present disclosure provides oligonucleotides, compositions, and methods thereof. Among other things, the present disclosure encompasses the recognition that structural elements of oligonucleotides, such as base sequence, chemical modifications (e.g., modifications of sugar, base, and/or internucleotidic linkages) or patterns thereof, conjugation with additional chemical moieties, and/or stereochemistry [e.g., stereochemistry of backbone chiral centers (chiral internucleotidic linkages)], and/or patterns thereof, can have significant impact on oligonucleotide properties and activities, e.g., knockdown ability, stability, delivery, etc. In some embodiments, the oligonucleotides decrease the expression, activity and/or level of a C9orf72 gene, including but not limited to, one comprising a repeat expansion, or a gene product thereof.
Type:
Application
Filed:
March 21, 2024
Publication date:
February 27, 2025
Inventors:
Chandra Vargeese, Zhong Zhong, Naoki Iwamoto, Jason Jingxin Zhang, Jean-Cosme Dodart, Yuanjing Liu, Pachamuthu Kandasamy, Sethumadhavan Divakaramenon, Genliang Lu, Subramanian Marappan