CRISPR gene editing: a revolution in progress

Abstract

CRISPR is a gene editing technique that has revolutionised research and has the potential to transform the treatment of many diseases. This article discusses the principles of the technique, its therapeutic applications and potential safety issues.

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“…CRISPR uses an enzyme that precisely cuts at a specific genetic sequence, which potentially alters the genome -such as switching a gene on or off. 11 "A large number of laboratories and companies are working feverishly on bringing CRISPR into the clinic," Professor Weissig says. Last November, the Medicines and Healthcare products Regulatory Agency (MHRA) authorised exagamglogene autotemcel, a CRISPR-based gene therapy for sickle cell disease and transfusion-dependent beta-thalassemia.…”

“…Many bacteria use CRISPR to defend themselves by degrading viral nucleic acids before they infect the cell. CRISPR uses an enzyme that precisely cuts at a specific genetic sequence, which potentially alters the genome – such as switching a gene on or off 11 . “A large number of laboratories and companies are working feverishly on bringing CRISPR into the clinic,” Professor Weissig says.…”