Therapeutic Approaches To Cure Genetic Disorders Cystic Fibrosis - A Review
DOI:
https://doi.org/10.53555/jaz.v40i01.4984Keywords:
Genetic Disorders, Cystic Fibrosis, Stem Cells, Genes, Mutations.Abstract
In this review, we describe the mechanism and methodology of gene editing therapy used to cure the cystic fibrosis. It is an autosomal recessive disorder that is, caused due to the mutation in the copies of CFTR gene. The gene editing therapy, here, used is, CRISPR/Cas9. This has been already clinically tested in animals and prokaryotes. Thus, far had an attempt to edit genes in the stem cells that originated from blood lineages. The advantage of the stem cells that are originated from blood is that, they can be selected, expanded, and returned to the body. Next trials are to be likely in liver. Studies suggested that the yeast has laid foundation for the genome editing. It could suggest that, gene editing would be possible if one could direct DNA double strand to a single site in the genome.in this review, we later discuss upon zinc-finger nucleases, which is Zinc-finger nucleases are artificial restriction enzymes generated by fusing a zinc finger DNA-binding domain to a DNA-cleavage domain. Zinc finger domains can be engineered to target specific desired DNA sequences and this enables zinc-finger nucleases to target unique sequences within complex genome. In this review, other than the information regarding the curing of cystic fibrosis by using genome editing, we will further discuss the challenges faced during the gene editing and also the information related to the gene editing therapy used in yeast.
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