“The pain was like being struck by lightning and being hit by a freight train at the same time,” shared Victoria Gray. New Scientist reflects on her journey: “Everything has changed for me now.”
Gray once endured debilitating symptoms of sickle cell disease, but in 2019, she found hope through CRISPR gene editing, a pioneering technology enabling precise modifications of DNA. By 2023, this groundbreaking treatment was officially recognized as the first approved CRISPR therapy.
Currently, hundreds of clinical trials are exploring CRISPR-based therapies. Discover the ongoing trials that signify just the beginning of CRISPR’s potential. This revolutionary tool is poised to treat a wide range of diseases beyond just genetic disorders. For example, a single CRISPR dose may drastically lower cholesterol levels, significantly reducing heart attack and stroke risk.
While still in its infancy regarding safety, there’s optimism that CRISPR could eventually be routinely employed to modify children’s genomes, potentially reducing their risk of common diseases.
Additionally, CRISPR is set to revolutionize agriculture, facilitating the creation of crops and livestock that resist diseases, thrive in warmer climates, and are optimized for human consumption.
Given its transformative capabilities, CRISPR is arguably one of the most groundbreaking innovations of the 21st century. Its strength lies in correcting genetic “misspellings.” This involves precisely positioning the gene-editing tool within the genome, akin to placing a cursor in a lengthy document, before making modifications.
Microbes utilize this genetic editing mechanism in their defense against other microbes. Before 2012, researchers identified various natural gene-editing proteins, each limited to targeting a single location in the genome. Altering the target sequence required redesigning the protein’s DNA-binding section, a process that was time-consuming.
However, scientists discovered that bacteria have developed a diverse range of gene-editing proteins that bind to RNA—a close relative of DNA—allowing faster sequence matching. Producing RNA takes mere days instead of years.
In 2012, Jennifer Doudna and her team at the University of California, Berkeley, along with Emmanuelle Charpentier from the Max Planck Institute for Infection Biology, revealed the mechanics of one such gene-editing protein, CRISPR Cas9. By simply adding a “guide RNA” in a specific format, they could target any desired sequence.
Today, thousands of variants of CRISPR are in use for diverse applications, all relying on guide RNA targeting. This paradigm-shifting technology earned Doudna and Charpentier the Nobel Prize in 2020.
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Source: www.newscientist.com
