The growing understanding of RNA functions and their crucial roles in diseases promotes
the application of various RNAs to selectively function on hitherto “undruggable” proteins …

With the improvement of gene vectors, the rise of chimeric antigen receptor T cell
immunotherapy and breakthroughs in the genome editing technology, gene therapy had …

Tau neuronal and glial pathologies drive the clinical presentation of Alzheimer's disease
and related human tauopathies. There is a growing body of evidence indicating that …

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

Abstract Neurodevelopmental Disorders (NDDs) encompass a broad spectrum of disorders,
linked because of their origins in brain developmental processes, including diverse …

Duchenne muscular dystrophy (DMD) is an X-linked recessive neuromuscular disorder that
causes debilitating muscle weakness and atrophy due to a loss of the dystrophin protein …

Gene therapy, recently frequently investigated, is an alternative treatment method that
introduces therapeutic genes into a cancer cell or tissue to cause cell death or slow down …

INTRODUCTION Alternative splicing is a posttranscriptional regulatory mechanism critical
for transcriptome and proteome diversity. By increasing complexity at the protein level …

Duchenne muscular dystrophy is a highly progressive muscle wasting disorder due to
primary abnormalities in one of the largest genes in the human genome, the DMD gene …

In 1995, we were the first to propose antisense oligonucleotide (ASO)-mediated exon-
skipping therapy for the treatment of Duchenne muscular dystrophy (DMD), a noncurable …