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 …

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 …

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 …

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 …

Introduction Gene therapy is becoming increasingly common in clinical practice, giving hope
for the correction of a wide range of human diseases and defects. The CRISPR/Cas9 …

Duchenne muscular dystrophy (DMD) was named more than 150 years ago. About four
decades ago, the DMD gene was discovered, and the reading frame shift was determined …

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 …

Muscle-directed gene therapy with adeno-associated viral (AAV) vectors is undergoing
clinical development for treating neuromuscular disorders and for systemic delivery of …