RNA base editing refers to the rewriting of genetic information within an intact RNA molecule
and serves various functions, such as evasion of the endogenous immune system and …

Amyotrophic lateral sclerosis (ALS) is the most common adult-onset motor neuron disease
classified as both a neurodegenerative and neuromuscular disorder. With a complex …

Due to their intrinsic nuclease resistance, mirror image L-oligonucleotides are being
increasingly employed in the development of biomedical research tools and therapeutics …

Further development of direct-acting antiviral agents against human SARS-CoV-2 infections
remains a public health priority. Here, we report that an antisense peptide-conjugated …

Splice modulating antisense oligomers (AOs) are increasingly used to modulate RNA
processing. While most are investigated for their use as therapeutics, AOs can also be used …

Single-stranded oligonucleotides (SSOs) are a rapidly expanding class of therapeutics that
comprises antisense oligonucleotides, microRNAs, and aptamers, with ten clinically …

Pathogenic variations in the fused in sarcoma (FUS) gene are associated with rare and
aggressive forms of amyotrophic lateral sclerosis (ALS). As FUS-ALS is a dominant disease …

Internucleotide phosphate diester bonds in unmodified oligonucleotides are rapidly
degraded by nucleolytic enzymes in cells or body fluids. This property excludes natural DNA …

This chapter provides an overview of mesyl phosphoramidate oligonucleotides (μ-
oligonucleotides), which incorporate mesyl phosphoramidate groups (MsPA, or μ) instead of …

Engineering new solutions for therapeutic benefit in Amyotrophic Lateral Sclerosis (ALS)
has proved a difficult task to accomplish. This is largely the reflection of complexities at …