A set of molecular technologies that enable scientists to precisely modify an organism’s DNA sequence by inserting, deleting, or replacing specific genetic material at targeted locations within the complex genome. This revolutionary tool has profound implications for treating monogenic and complex genetic disorders, advancing fundamental biological research, and potentially influencing human longevity and healthspan. Clinical applications are rapidly emerging, requiring careful ethical and safety consideration.
Origin
The term gene editing describes the action of making precise, intentional changes to the foundational blueprint of life, the genome. The modern era of gene editing was largely ushered in by the development of highly programmable nucleases, particularly the CRISPR-Cas9 system, which significantly simplified and made the process highly efficient. This technology built upon decades of foundational research into the cell’s natural DNA repair mechanisms.
Mechanism
The most common mechanism, utilizing the CRISPR-Cas9 system, involves a guide RNA molecule directing the Cas9 enzyme to a specific, complementary sequence in the DNA double helix. Cas9 then acts as molecular scissors, creating a double-strand break at that precise site. The cell’s natural DNA repair pathways, either non-homologous end joining (NHEJ) or homology-directed repair (HDR), are subsequently utilized to introduce or correct the desired genetic modification.
Advanced gene editing for male infertility raises ethical questions about systemic hormonal impacts, long-term health, and the intergenerational responsibilities of altering human lineage.
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