CRISPR-based drugs can theoretically manipulate any genetic target. In practice, however,
these drugs must enter the desired cell without eliciting an unwanted immune response, so …

The discovery of clustered regularly interspaced short palindromic repeats (CRISPR)
genome editing technology opened the door to provide a versatile approach for treating …

CRISPR-Cas9 has emerged as a powerful technology that relies on Cas9/sgRNA
ribonucleoprotein complexes (RNPs) to target and edit DNA. However, many therapeutic …

Genome editing therapy for Duchenne muscular dystrophy (DMD) holds great promise,
however, one major obstacle is delivery of the CRISPR-Cas9/sgRNA system to skeletal …

Mutations in RNA binding motif protein 20 (RBM20) are a common cause of familial dilated
cardiomyopathy (DCM). Many RBM20 mutations cluster within an arginine/serine-rich (RS …

Genome editing technologies operate by inducing site-specific DNA perturbations that are
resolved by cellular DNA repair pathways. Products of genome editors include DNA breaks …

Duchenne muscular dystrophy (DMD) is a fatal muscle disease caused by the lack of
dystrophin, which maintains muscle membrane integrity. We used an adenine base editor …

According to Darwin's theory, endless evolution leads to a revolution. One such example is
the Clustered Regularly Interspaced Palindromic Repeats (CRISPR)–Cas system, an …

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

Lipid nanoparticles (LNPs) have revolutionized the field of drug delivery through their
applications in siRNA delivery to the liver (Onpattro) and their use in the Pfizer-BioNTech …