An Examination of Gene Editing and Gene Therapy in Genetic Diseases
DOI:
https://doi.org/10.58445/rars.834Keywords:
Gene Editing, Gene Therapy, Genetic DiseasesAbstract
The field of gene editing and therapy has helped the scientific community understand treatment
options related to different genetic diseases such as ADA-SCID, Huntington's disease, and
cystic fibrosis. Research has shown that CRISPR, a gene editing tool, has helped in treating
genetic diseases that currently do not have conventional treatments available. Although being a
greatly beneficial tool, CRISPR comes with its limitations such as leading to a possible risk of
cancer through off-target edits. Gene therapy is a direct approach to treating genetic diseases,
such as ADA-SCID, through either in-vivo or ex-vivo applications. This paper analyzes the
application of gene editing and gene therapy for ADA-SCID, Huntington's disease, and cystic
fibrosis, while also considering the ethical implications.
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