RNA base editing refers to the rewriting of genetic information within an intact RNA molecule
and serves various functions, such as evasion of the endogenous immune system and …

The Clustered Regularly Interspaced Short Palindromic Repeat (CRISPR) and its
accompanying protein (Cas9) are now the most effective, efficient, and precise genome …

Cardiovascular disease remains the leading cause of morbidity and mortality in the
developed world. In recent decades, extraordinary effort has been devoted to defining the …

Regulatory T cells are critical for maintaining immune tolerance. Recent studies have
confirmed their therapeutic suppressive potential to modulate immune responses in organ …

CRISPR offers new hope for many patients and promises to transform the way we think of
future therapies. Ensuring safety of CRISPR therapeutics is a top priority for clinical …

Genome editing has been a transformative force in the life sciences and human medicine,
offering unprecedented opportunities to dissect complex biological processes and treat the …

Recombinant adeno-associated virus (AAV) is a promising delivery vehicle for in vivo gene
therapy and has been widely used in> 200 clinical trials globally. There are already several …

Duchenne muscular dystrophy (DMD) is a lethal neuromuscular disease caused by
mutations in the X-linked dystrophin (DMD) gene. Exon deletions flanking exon 51, which …

Upon viral infection of the liver, CD8+ T cell responses may be triggered despite the immune
suppressive properties that manifest in this organ. We sought to identify pathways that …

Alzheimer's disease (AD) is the most burdensome aging‐associated neurodegenerative
disorder, and its treatment encounters numerous failures during drug development …