High efficacy, selectivity and cellular targeting of therapeutic agents has been an active area
of investigation for decades. Currently, most clinically approved therapeutics are small …

Introduction lncRNAs are major players in regulatory networks orchestrating multiple cellular
functions, such as 3D chromosomal interactions, epigenetic modifications, gene expression …

With over 15 FDA approved drugs on the market and numerous ongoing clinical trials, RNA
therapeutics, such as small interfering RNAs (siRNAs) and antisense oligonucleotides …

Duchenne muscular dystrophy (DMD) is a severe, progressive, muscle-wasting disease,
characterized by progressive deterioration of skeletal muscle that causes rapid loss of …

Introduction It is established that the exon-skipping approach can restore dystrophin in
Duchenne muscular dystrophy (DMD) patients. However, dystrophin restoration levels are …

Antisense oligonucleotide (ASO)-mediated exon skipping can restore the open reading
frame of dystrophin transcripts for Duchenne muscular dystrophy (DMD) patients. This …

Duchenne muscular dystrophy (DMD) is a devastating disease that results in life-limiting
complications such as loss of skeletal muscle function as well as respiratory and cardiac …

Rare diseases (RD) affect a small number of people compared to the general population
and are mostly genetic in origin. The first clinical signs often appear at birth or in childhood …

Duchenne muscular dystrophy (DMD) is a progressive X-linked disease caused by
mutations in the DMD gene that prevent the expression of a functional dystrophin protein …

Duchenne muscular dystrophy (DMD) is an X-linked genetic disease primarily affecting boys
causing loss of the dystrophin protein, ultimately leading to muscle wastage and death by …