RNA-based therapies have catalyzed a revolutionary transformation in the biomedical
landscape, offering unprecedented potential in disease prevention and treatment. However …

RNA‐based therapeutics have the potential to revolutionize the treatment and prevention of
human diseases. While early research faced setbacks, it established the basis for …

Targeted protein degradation (TPD) has been established as a viable alternative to
attenuate the function of a specific protein of interest in both biological and clinical contexts …

Chemical modification of nucleotides can improve the metabolic stability and target
specificity of oligonucleotide therapeutics, and alkylphosphonates have been employed as …

Antisense oligonucleotides (ASOs) are incredibly versatile molecules that can be designed
to specifically target and modify RNA transcripts to slow down or halt rare genetic disease …

Argonaute protein is associated with post-transcriptional control of cytoplasmic gene
expression through miRNA-induced silencing complexes (miRISC). Specific cellular and …

Chemical modifications are necessary to ensure the metabolic stability and efficacy of
oligonucleotide-based therapeutics. Here, we describe analyses of the α-(l)-threofuranosyl …

More than 25 years after its discovery, the post-transcriptional gene regulation mechanism
termed RNAi is now transforming pharmaceutical development, proved by the recent FDA …

siRNA therapeutics have gained extensive attention, and to date six siRNAs are approved
for clinical use. Despite being investigated for the treatment of metabolic, cardiovascular …

Nanoparticle-based delivery systems have contributed to the recent clinical success of RNA
therapeutics, including siRNA and mRNA. RNA delivery using polymers has several distinct …