International Journal of Scientific and Research Publications

IJSRP, Volume 11, Issue 1, January 2021 Edition [ISSN 2250-3153]


A review of the therapeutic potential, prospects, and challenges of CRISPR/Cas9 genome editing in the treatment of Sickle Cell Disease (SCD)
      Arundhati Chaudhary, Barsha Kumari, Meghna Choudhury, Shubham Singh, Dr. Ramneet Kaur
Abstract: 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 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.

Reference this Research Paper (copy & paste below code):

Arundhati Chaudhary, Barsha Kumari, Meghna Choudhury, Shubham Singh, Dr. Ramneet Kaur (2021); A review of the therapeutic potential, prospects, and challenges of CRISPR/Cas9 genome editing in the treatment of Sickle Cell Disease (SCD); International Journal of Scientific and Research Publications (IJSRP) 11(1) (ISSN: 2250-3153), DOI: http://dx.doi.org/10.29322/IJSRP.11.01.2021.p10983
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