Since its inception, antisense oligonucleotides (ASOs) have been extensively used in
functional genomics. Recent advancements in chemical modification and delivery …

Rare diseases affect almost 500 million people globally, predominantly impacting children
and often leading to significantly impaired quality of life and high treatment costs. While …

Spinal muscular atrophy (SMA) is a severe genetic disorder characterized by the loss of
motor neurons, leading to progressive muscle weakness, loss of mobility, and respiratory …

Alpha-synuclein (αSyn) aggregates are the primary component of Lewy bodies, which are
pathological hallmarks of Parkinson's disease (PD). The toxicity of αSyn seems to increase …

Spinal muscular atrophy (SMA) is a severe neuromuscular disorder that is caused by
mutations in the survival motor neuron 1 (SMN1) gene, hindering the production of …

Introduction Antisense oligonucleotide (ASO) was established as a viable therapeutic option
for genetic disorders. ASOs can target RNAs implicated in various diseases, including …

Chronic kidney disease (CKD) is one of the leading causes of mortality worldwide because
of kidney failure and the associated challenges of its treatment including dialysis and kidney …

Casimersen (AMONDYS 45TM) is an antisense oligonucleotide of the phosphorodiamidate
morpholino oligomer subclass developed by Sarepta therapeutics. It was approved by the …

Currently, four kinds of phosphorodiamidate morpholino oligomers (PMOs), such as
viltolarsen, have been approved for the treatment of Duchenne muscular dystrophy (DMD); …

Abstract Duchenne Muscular Dystrophy (DMD) is a devastating X-linked genetic disorder
characterized by progressive muscle degeneration due to mutations in the dystrophin gene …