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Mesyl Phosphoramidate Oligonucleotides as Potential Splice-Switching Agents: Impact of Backbone Structure on Activity and Intracellular Localization

Research output: Contribution to journal › Article › peer-review

DOI

https://doi.org/10.1089/nat.2020.0860
Final published version

Suzan M. Hammond
Olga V. Sergeeva
Pavel A. Melnikov
Larissa Goli
Jessica Stoodley
Timofei S. Zatsepin
Dmitry A. Stetsenko
Matthew J.A. Wood

Original language	English
Pages (from-to)	190-200
Number of pages	11
Journal	Nucleic Acid Therapeutics
Volume	31
Issue number	3
DOIs	https://doi.org/10.1089/nat.2020.0860
Publication status	Published - Jun 2021

Research areas

2′-methoxyethyl, intracellular distribution, nusinersen, spinal muscular atrophy, splice switching

OECD FOS+WOS

3.01 BASIC MEDICAL RESEARCH
1.06 BIOLOGICAL SCIENCES
1.06.CQ BIOCHEMISTRY & MOLECULAR BIOLOGY
3.01.TU PHARMACOLOGY & PHARMACY
3.01.QA MEDICINE, RESEARCH & EXPERIMENTAL

ID: 34033282