When the drug fomivirsen was approved by the FDA in 1998 for the treatment of cytomegalovirus retinitis in patients with HIV/AIDs, it was hailed as a milestone in drug discovery because it was the ...

Researchers found that adding a newly developed modified sugar, BNAP-AEO, to gapmer antisense oligonucleotides (ASOs) increased their affinity for target RNAs, thus significantly enhancing their ...

Researchers from Japan investigated how ASOs administered locally into the brains of mice models for Parkinson’s disease can help prevent the formation and spread of harmful aSyn aggregates through ...

Stargardt disease type 1 (STGD1) is an inherited retinal recessive disease caused by biallelic variants in the ABCA4 gene. One of the recurrent variants is located at the exon-intron junction of exon ...

Please provide your email address to receive an email when new articles are posted on . Wave Life Sciences has announced positive results from its phase 1b/2a clinical trial of WVE-003, a potential ...

AMX0114 is an investigational antisense oligonucleotide targeting calpain-2, a calcium-activated protease believed to play a role in ALS disease progression. The Food and Drug Administration (FDA) has ...

Delivering cutting-edge peer-reviewed research on using nucleic acids and related compounds to alter gene expression for therapeutic purposes. A new article in the peer-reviewed journal Nucleic Acid ...

Ludwig-Maximilians-Universität München-led researchers have induced a greater than 60% reduction in seizure frequency in a preterm infant with early-onset SCN2A developmental and epileptic ...

By improving gait stability, Ionis’ zilganersen could be “potentially disease modifying,” according to analysts at William ...

TMDU researchers demonstrate proof of concept of antisense nucleic acid therapy to prevent the spread of α-synuclein pathologies in synucleinopathies. Parkinson's disease (PD), as well as many other ...