At present, the idea of genome modification has revolutionized the modern therapeutic
research era. Genome modification studies have traveled a long way from gene …

Hemophilia is an X-linked inherited bleeding disorder, resulting from defects in the F8
(hemophilia A) or F9 (hemophilia B) genes. Persons with hemophilia have bleeding …

Duchenne muscular dystrophy (DMD) is a monogenic disorder and a candidate for
therapeutic genome editing. There have been several recent reports of genome editing in …

Extensive preclinical research over the past 30 years has culminated in the recent regulatory
approval of several gene therapy products for hemophilia. Based on the efficacy and safety …

Cancer is one of the most leading causes of death and a major public health problem,
universally. According to accumulated data, annually, approximately 8.5 million people died …

The rapid development of CRISPR–Cas technologies brought a personalized and targeted
treatment of genetic disorders into closer reach. To render CRISPR-based therapies precise …

Clustered regularly interspaced short palindromic repeats‐associated protein
(CRISPR/Cas9), an adaptive microbial immune system, has been exploited as a robust …

CRISPR/Cas9 system exploits the concerted action of Cas9 nuclease and programmable
single guide RNA (sgRNA), and has been widely used for genome editing. The Cas9 …

The liver plays a protective role in myocardial infarction (MI). However, very little is known
about the mechanisms. Here, we identify mineralocorticoid receptor (MR) as a pivotal nexus …

Hemophilia B (HB) is a life-threatening inherited disease caused by mutations in the FIX
gene, leading to reduced protein function and abnormal blood clotting. Due to its monogenic …