Summary The coronavirus disease 2019 (COVID-19) that is wreaking havoc on worldwide
public health and economies has heightened awareness about the lack of effective antiviral …

Peptide-oligonucleotide conjugates (POCs) represent one of the increasingly successful
albeit costly approaches to increasing the cellular uptake, tissue delivery, bioavailability …

Duchenne muscular dystrophy (DMD), a fatal X-linked recessive disorder, is caused mostly
by frame-disrupting, out-of-frame deletions in the dystrophin (DMD) gene. Antisense …

Low CFTR mRNA expression due to nonsense-mediated mRNA decay (NMD) is a major
hurdle in developing a therapy for cystic fibrosis (CF) caused by the W1282X mutation in the …

Antisense oligonucleotide (ASO) technology has become an attractive therapeutic modality
for various diseases, including Mendelian disorders. ASOs can modulate the expression of a …

Exon skipping is a therapeutic approach that is feasible for various genetic diseases and
has been studied and developed for over two decades. This approach uses antisense …

Introduction: Limb-girdle muscular dystrophies (LGMDs) encompass a clinically
heterogeneous group of rare, genetic progressive muscle disorders presenting with …

The click azide= alkyne 1, 3-dipolar cycloaddition (click chemistry) has become the
approach of choice for bioconjugations in medicinal chemistry, providing facile reaction …

A series of 2′-deoxy and novel 2′-O-methyl and 2′-O-(2-methoxyethyl)(2′-MOE)
oligonucleotides with internucleotide methanesulfonyl (mesyl, μ) or 1-butanesulfonyl (busyl …

Precision medicine in oncology has made significant progress in recent years by approving
drugs that target specific genetic mutations. However, many cancer driver genes remain …