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Angelman Syndrome Therapies Show Positive Results in Early Phase Clinical Trials

• Perspectives on Current Science

September 24, 2024

Encouraging Progress in the Hunt for a Huntington’s Treatment

• Perspectives on Current Science

September 11, 2024

mRNA Nuclear Clustering Leads to a Difference in Mutant Huntingtin mRNA and Protein Silencing by siRNAs In Vivo

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August 28, 2024

Sequence- and Structure-Dependent Cytotoxicity of Phosphorothioate and 2′-O-Methyl Modified Single-Stranded Oligonucleotides

Authors: Laura V. Croft  Mark Fisher, Tabassum Khair Barbhuiya, Serene El-Kamand, Samuel Beard, Aleksandra Rajapakse, Roland Gamsjaeger, Liza Cubeddu, Emma Bolderson, Ken O’Byrne , Derek Richard derek.richard@qut.edu.au, and Neha S. Gandhi Published Online: 17 June 2024 Abstract Single-stranded oligonucleotides (SSOs) …

• Open Access NAT
• Perspectives on Current Science

August 22, 2024

Nucleic Acid Therapeutics: Successes, Milestones, and Upcoming Innovation

• Open Access NAT
• Perspectives on Current Science