First-ever in-body CRISPR human trial performed by Clinicians

The clinical trial at the Oregan Health & Science University was performed to treat a blindness-causing gene mutation

By Faisal Khan | 18 March 2020

Andreas Lauer, M.D., right, performs the first-ever in vivo CRISPR gene edit procedure for the BRILLIANCE clinical trial. (OHSU / Kristyna Wentz-Graff)

The revolutionary medical technology CRISPR has come a long way ever since it was developed back in 2012. Although there has been some controversy attached to the use of this futuristic medical tool by a Chinese scientist, the results of some of the other trials have been nothing less than miraculous. The tool basically acts as a pair of DNA scissors to modify the genes by replacing the defected ones in the double helix of the DNA.

Researchers have actually been at work to modify and improve the technique to make it more useful and efficient. The latest version of the tool dubbed as CRISPR 2.0 improves on the original technique—having the capability of adding or deleting long strands of DNA, but without breaking the DNA helix. More recently, another efficient version called INTEGRATE was introduced providing a gentle and less invasive editing technique.

Chinese scientists have been at the forefront of testing this technology. They were in the news recently, with the claim of using the CRISPR tool for trying to treat an HIV & Cancer infected male. While these claims were treated with some skepticism, the first human trial of CRISPR outside China proved the effectiveness of the innovative medical tool.

However, most of these studies and trials were done by removing the cells from the patients, editing them with CRISPR and then returning the fixed cells back into the body. Now, for the first time, Clinicians have conducted an in-body CRISPR editing in a trial study. Scientists at the Oregon Health and Science University (OHSU) tested the CRISPR tool to treat a genetic mutation that causes blindness.

“Being able to edit genes inside the human body is incredibly profound. Beyond potentially offering treatment for a previously untreatable form of blindness, in vivo gene editing could also enable treatments for a much wider range of diseases.”

~ Mark Pennesi, Lead scientist in the trial

The human trial conducted at the OHSU’s eye institute was sponsored by private companies Allergan plc and Editas Medicine. The technique targeted a rare condition Leber congenital amaurosis type 10 (LCA10)—triggered by a genetic mutation, it affects the retina, causing people to be either born blind or lose their sight in the earlier years of their life.

CRISPR seeks to repair the mutations which occur in the gene called CEP290, which causes this inherited blindness. Clinicians injected the CRISPR-loaded medicine directly into photoreceptor cells behind the retina, where it gets to work of removing and replacing the faulty gene.

This was the first of its kind treatment and so far only one patient has been trialed. A bigger pool of 18 participants is expected to take part, which will present a clear picture of the effectiveness and safety of the treatment, apart from figuring out how the body reacts to the treatment. With each additional study though, CRISPR continues to present itself as a promising tool in Healthtech.

Reprinted with permission from the author.

Faisal Khan is a prolific Canada-based tech blogger and influencer. He is the founder and editor of the Technicity publication which focuses on technical, scientific and financial knowledge sharing. Follow him on Twitter @fklivestolearn.

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