Of the recent advances in gene therapy, the CRISPR/Cas genome editing technique is noteworthy for its specificity and applications. CRISPR/Cas enables non-homologous end joining, single-base exchanges, and homology-directed repair in mammalian cells. For genome editing, Cas nuclease comes from Streptococcus pyogenes (SpCas9) and is a part of the type II CRISPR system. The CRISPR/Cas system has numerous applications such as therapeutic genome editing and monogenic diseases treatment .
It has the potential to be used directly in patients (in vivo) or in human cells (in vitro). The CRISPR/Cas systems are used to research target genes in genome modification, transcription, splicing, and epigenetic regulation. These systems have been applied in research settings to investigate and treat genetic diseases, infectious diseases, cancers, and immunological diseases. Due to the simplicity and…
