Most of the human genome encodes RNAs that do not code for proteins. These non-coding
RNAs (ncRNAs) may affect normal gene expression and disease progression, making them …

Antisense transcription, which was initially considered by many as transcriptional noise, is
increasingly being recognized as an important regulator of gene expression. It is …

Background Mutations that disrupt the open reading frame and prevent full translation of
DMD, the gene that encodes dystrophin, underlie the fatal X-linked disease Duchenne …

Synthetic nucleic acids are commonly used laboratory tools for modulating gene expression
and have the potential to be widely used in the clinic. Progress towards nucleic acid drugs …

Duchenne muscular dystrophy (DMD) is a degenerative muscle disease caused by genetic
mutations that lead to the disruption of dystrophin in muscle fibers. There is no curative …

CRISPR gene editing has revolutionized biomedicine and biotechnology by providing a
simple means to engineer genes through targeted double-strand breaks in the genomic …

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

Skeletal muscle disorders have posed great threats to health. Selective delivery of drugs
and oligonucleotides to skeletal muscle is challenging. Aptamers can improve targeting …

Almost all protein-coding genes are spliced and their majority is alternatively spliced.
Alternative splicing is a key element in eukaryotic gene expression that increases the coding …

Ninety‐four percent of human genes are discontinuous, such that segments expressed as
mRNA are contained within exons and separated by intervening segments, called introns …