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Genome Editing - Shaping the Future of Gene Therapy


 

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Introduction to Gene Editing

A scientist working on his PhD in Spain identified genomic sequences in archaea that were repeated up to 600 times in a row in the early 1990s. In the late 1980s, Japanese scientists made a similar discovery in bacteria. Later research discovered that these repeats are part of a prokaryotic immune system. They are employed to retain genetic information on bacterial viruses (bacteriophages) that the organism and its ancestors have been exposed to, as well as ready the organism to defend itself against that invader in the future. Few people would have predicted that this discovery, known as CRISPR, would lead to the current frenzy in mammalian genome editing.


Since the 1970s, genetic engineering has been used to introduce new genetic components into organisms. The random manner with which the DNA is inserted into the host's genome has been a downside of this method, impairing or altering other genes inside the organism.


However, various ways for targeting inserted genes to specific places within an organism's genome have been discovered. It has also made it possible to modify specific sequences inside a genome while reducing off-target effects. Before the advent of the current nuclease-based gene-editing platforms, genome editing was pioneered in the 1990s, but its utility was limited by poor editing efficiency.


Emmanuelle Charpentier of the Max Planck Unit for the Science of Pathogens and Jennifer Doudna of the University of California, Berkeley were jointly awarded the Nobel Prize in Chemistry in 2020 to develop the CRISPR/Cas9 genetic scissors, which have revolutionized genome editing.


What is Genome/Gene Editing?

Genome editing, also known as genome engineering or gene editing, involves inserting, deleting, modifying, or replacing DNA in a living organism's genome. Unlike earlier genetic engineering approaches that randomly inserted genetic material into a host genome, genome editing focuses insertions to specified sites. Editing DNA can alter physical characteristics such as eye colour and disease risk. To achieve so, scientists employ a variety of techn