Unveiling sequence-agnostic mixed-chemical modification patterns for splice-switching oligonucleotides using the NATURA platform

Unveiling sequence-agnostic mixed-chemical modification patterns for splice-switching oligonucleotides using the NATURA platform

Chemical optimization of ribose has significantly advanced nucleic acid therapeutics (NATs) by improving the stability, specificity, and safety of therapies like small interfering RNAs, CRISPR-Cas9 guide RNAs, and GAPmers. Recent research has extended this approach to splice-switching oligonucleotid...

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Bibliographic Details
Main Authors: Tommaso Tabaglio, Taniya Agarwal, Wei Yuan Cher, Jin Rong Ow, Ah Keng Chew, Priscila Yun Qian Sun, Raja Sekhar Reddy Gurrampati, Hongfang Lu, Praveena Naidu, Hong Kai Ng, Xavier Le Guezennec, Shi Yan Ng, Manikandan Lakshmanan, Ernesto Guccione, Keng Boon Wee
Format: Article
Language:English
Published: Elsevier 2025-03-01
Series:Molecular Therapy: Nucleic Acids
Subjects:
MT: Oligonucleotides: Therapies and Applications
antisense oligonucleotides
nucleic acid therapeutics
reporter system
nucleic acid chemical modifications
mixmers
Online Access:http://www.sciencedirect.com/science/article/pii/S2162253124003093
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