As sickle cell disease is a well-known genetic disorder of point mutation, it is considered a leading candidate for geneediting therapies. Studies published in 2016 described a successful proof-of-concept in treating sickle cell disease in mice using the CRISPR-Cas9 gene-editing tool. CRISPR -Cas9 is a programmable RNA-guided DNA endonuclease, which has been gaining significant attention over the last decade due to its ability to treat genetic disorders such as sickle cell disease. Guided by RNA strand, the Cas9 nuclease-originally isolated from bacteria can be programmed to cut a target DNA sequence and modified by inserting, deleting, or replacing it with the normal copy of the genetic sequence. Though successfully demonstrated in mice species, this genome editing tool is still in a very nascent stage concerning utilization in the human population. Through this review paper, we analyze the scope, possibilities of CRISPR-Cas9 as a potential therapeutic tool in the management of sickle cell disease.
scite is a Brooklyn-based organization that helps researchers better discover and understand research articles through Smart Citations–citations that display the context of the citation and describe whether the article provides supporting or contrasting evidence. scite is used by students and researchers from around the world and is funded in part by the National Science Foundation and the National Institute on Drug Abuse of the National Institutes of Health.