A transformative technology for gene editing - clustered regularly interspaced short palindromic repeats (CRISPR) -- has become ubiquitous, at least in research laboratories, where scientists efficiently adopted CRISPR technology to manipulate genes of interest. Still, applying it for humans is associated with greater risks and faces ethical issues. However, alongside a too reckless experiment of a Chinese scientist who edited babies’ genomes, plenty of companies have made well-thought progress toward transferring gene editing technologies into the clinic. Here we make a brief but pithy review on up-to-date gene editing approaches and start-up companies active in this area.
CRISPR-Cas9 begins to demonstrate positive results in treating inherent diseases in clinical trials: CTX001 developed by CRISPR Therapeutics showed positive results in patients with beta-thalassemia and sickle cell disease, and Editas Medicine’s promising drug for editing retinal gene defect is now in early-stage clinical trials. The third notable player is Intellia Therapeutics which is focusing on advancing methods of in vivo delivery to the liver. Intellia plans to commence trials of their CRISPR/Cas9 therapy in 2020, which is going to become the first systemically delivered investigational drug entered the clinic.
CRISPR/Cas9 has been borrowed from bacteria that utilizes it to inactivate viruses by cutting their DNA. Cas9 is a nuclease that makes double-strand cuts and is delivered to the right place in the genome by a guide RNA. CRISPR/Cas9 can be used to disable a gene (gene knock-out), make a deletion or correct a gene by adding a DNA template.
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