Gene Editing Technology : Overview

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Gene-Editing Technology: Overview

a. Goals

In order to explore the ethical landscape of genome editing, it is first necessary to discuss the range of methods, the differing goals, and the recent advances in this biotechnology. According to the director of the NIH, Dr. Francis Collins, genome editing is an “area of research seeking to modify genes of living organisms to improve our understanding of gene function and advance potential therapeutic applications to correct genetic abnormalities.” More specifically, different gene-editing technologies have the goals of “precise manipulation of gene sequences in their natural chromosomal context and addition of transgenes to specific genomic loci.” Genome editing technologies have a wide range
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In the lab, scientists program these nucleases to recognize “site-specific DNA-binding domains,” which come in various forms. In the cell, the nucleases work by creating double-strand breaks (DSBs) at specific sites, which activates the normal DNA repair machinery of the cell to put the strand back together in a new way. In effect, this technology creates a pathway to “disrupt a gene or excise segments of genomes.” Alternatively, the double-strand breaks can induce recombination at higher rates, such that a “homologous donor template can be delivered to the cells along with the nuclease to target gene addition to that site or make small substitutions to gene sequences.”
Recent refinements to this technology have made it more accessible to scientists everywhere, as newer methods allow for “enhanced nuclease performance, accelerated nuclease assembly, and lowered the cost of genome editing.” Furthermore, these synthetic nucleases are able to target different cells,
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