Genome Editing – A New Class of Drugs Is Emerging!

Containing 3 billion nucleotides, our DNA contains approximately 22 thousand genes . These genes, which encode proteins and are called the meaningful part of DNA, correspond to approximately %2 of our DNA, and the expression of all genes is called the genome . Many diseases result from these genes undergoing mutation. Cancer is one of them. Some genetic diseases result from a single-point mutation, while many chronic diseases involve multiple mutations. A small proportion of these mutations are inherited (passed down from parents), while most are sporadic (arising from environmental factors and aging). Whether inherited or sporadic, one of medicine’s main goals is to correct genetic mutations resulting in disease. For this purpose, the idea of genome editing was born.

Below, there is an infographic covering the definition, history, diseases in which genome editing has been tested, and its limitations (note: the videos have Turkish subtitles added).

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Genome editing has now entered clinical trials: applications include correcting changes that cause single-gene diseases and developing chimeric antigen receptor (CAR) T-cell therapy and cell-based regenerative medicines. 

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There are many genome-editing techniques. Among them, CRISPR is the newest and most promising method.

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