Журнал протеомики и биоинформатики
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A Review of the Therapeutic Potential, Prospects and Challenges of CRISPR/Cas9 Genome Editing in the Treatment of Sickle Cell Disease

Arundhati Chaudhary

As sickle cell disease is a well-known genetic disorder of point mutation, it is considered a leading candidate for gene editing therapies. Studies published in 2016 described a successful proof-of-concept in treating sickle cell disease into mice using the CRISPR-Cas9 gene editing tool.

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 cells 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 with respect to utilization in 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.