Journal of Proteomics & Bioinformatics
Open Access
ISSN: 0974-276X
ISSN: 0974-276X
Department of Proteomics, National Dairy Research Institute, Karnal, India
Review Article
A Review of the Therapeutic Potential, Prospects and Challenges of CRISPR/Cas9 Genome Editing in the Treatment of Sickle Cell Disease
Author(s): 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.. View More»