Antisense oligonucleotides (ASOs) were first discovered to influence RNA processing and
modulate protein expression over two decades ago; however, progress translating these …

Antisense oligonucleotides (ASOs) represent a new and highly promising class of drugs for
personalized medicine. In the last decade, major chemical developments and improvements …

Splice-switching oligonucleotides (SSOs) are short, synthetic, antisense, modified nucleic
acids that base-pair with a pre-mRNA and disrupt the normal splicing repertoire of the …

Here, we discuss three RNA-based therapeutic technologies exploiting various
oligonucleotides that bind to RNA by base pairing in a sequence-specific manner yet have …

Background: In 1991, the first world survey of neuromuscular disorders (NMDs) was
published in the peer reviewed literature. Since then, diagnostics have been greatly …

A large and complex gene on the X chromosome encodes dystrophin. Many mutations have
been described in this gene, most of which affect the expression of the muscle isoform, the …

Duchenne muscular dystrophy (DMD) is a severe, progressive, and ultimately fatal disease
of skeletal muscle wasting, respiratory insufficiency, and cardiomyopathy. The identification …

Conceptual simplicity, the possibility of rational design, relatively inexpensive cost, and
developments in the sequencing of human genome have led to the use of short fragments of …

Current molecular genomic approaches to human genetic disorders have led to an
explosion in the identification of the genes and their encoded proteins responsible for these …

Antisense‐mediated exon skipping aiming for reading frame restoration is currently a
promising therapeutic application for Duchenne muscular dystrophy (DMD). This approach …