A first for Prime Editing

It had never happened before that a company decided to submit a commercial authorization request for a therapy tested on only two people.

We do not know the name of the teenager from Vancouver who, a year ago, became the first person in the world to receive a treatment based on a genetic correction approach similar to Word’s “find and replace.” What we do know is that before becoming a pioneer patient, even a common cold represented a serious threat to him. The father of the technique known as prime editing, David Liu, now describes him as “healthy, stable, and living with a functioning immune system.” Seeing him on skis in the the snow in the photo published by the Canadian Institutes of Health, is worth more than many words. The American National Institutes of Health, for their part, confirm that the second patient treated is also doing well.

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CRISPR N=1 in 101 seconds

Petros Giannikopoulos, director of the clinical laboratory at the Innovative Genomics Institute at the University of California, Berkeley, explains in 101 seconds the bespoke treatment developed in record time for a very special patient, whom we have mentioned many times here on CRISPeR Frenzy: Baby KJ. The video was shot by Sean Patrick Farrell.

Edited plants in the EU: countdown to reform

A little more patience. The European Parliament’s final vote, originally scheduled for March, has been postponed to late April, but the finish line is in sight. We should finally have rules that distinguish true transgenic plants (containing foreign DNA) from edited plants that do not contain extra genes and are indistinguishable from traditional breeding programs.

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CRISPR hemp, how and why

Credit: Un. of Wisconsin-Madison Crop Innovation Center

In a book published several years ago titled The Botany of Desire, Michael Pollan devoted a chapter to the evolution of cannabis. When our ancestors began cultivating hemp in the Neolithic period, they were interested in its fibers, but they were soon won over by its special effects. Perhaps it wasn’t we who domesticated cannabis, it may have been cannabis that domesticated us. The question is: why did evolution favor the production of psychoactive substances? Perhaps cannabinoids help the plant protect itself from radiation damage or pests. Or maybe, Pollan suggests, they enabled hemp to win the favor of human beings, who then actively cultivated it. Now evolution continues in labs, also with the help of gene editing, including efforts to eliminate those psychoactive substances from varieties used for therapeutic purposes. The latest news is that two new varieties have recently been deregulated in the United States.

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The first CRISPR therapy works, but adoption is slow

Two years after Casgevy received commercial approval, only around sixty people with sickle cell disease or thalassemia have been able to benefit from it, due to a technical hurdle that the next generation of treatments will attempt to overcome

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CRISPR: hitting the genetic bullseye

Imagine throwing a trillion darts and having every single one hit the bullseye. Achieving this level of precision in gene editing would require highly intelligent delivery of the molecular machinery for DNA repair (CRISPR or one of its variants) into patients’ bodies, reaching only defective cells while bypassing healthy tissues. The benefits would be substantial: maximum therapeutic efficiency, zero waste, and reduced risks in terms of toxicity, immunogenicity, and unwanted mutations. How can such precise targeting be achieved? By acting on multiple levels, explain Jennifer Doudna and three researchers from her Innovative Genomics Institute. See their review article, Targeted delivery of genome editors in vivo in Nature Biotechnology.

ISAAA: a window on plant gene editing

The blog also aims to highlight authoritative sources to keep readers up to date. ISAAA is an international non-profit organization dedicated to documenting applications of agricultural biotechnology and providing accurate information. We are pleased to recommend its supplement focused on gene editing. The latest issue covers a high-yield rice variety ready for cultivation in India, another drought-resistant rice developed in Indian laboratories, a genetically modified potato designed to reduce waste in Chile, and, finally, a scanner developed in Berkeley to identify gene switches in plants. Enjoy reading!

Gene editing’s new bet on PKU

Jennifer Doudna and Fyodor Urnov have founded a company that will take on the challenge of editing rare diseases, starting with phenylketonuria.

The problem is well known: many diseases are theoretically treatable by correcting the corresponding genetic defects with the help of CRISPR tools, but doing so risks being difficult or even impossible because of regulatory rigidity and economic unsustainability. The case of the first newborn treated with a bespoke therapy developed in record time (KJ Muldoon) was a splendid proof of principle. But it left many parents of children with rare diseases asking: when will it be our turn? At the same time, specialists have long been asking: will investors return to viewing gene editing as a profitable approach worth investing in?

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A fresh graphic take on gene editing

Tired of illustrating articles and PowerPoint presentations on genome editing with the usual genetic scissors and Swiss Army knives? You might not know that the Innovative Genomics Institute, founded by CRISPR inventor Jennifer Doudna, offers images created by its graphic designers free of charge for educational and non-commercial use. Take a look at the Science × Art section of their website and their illustrated glossary.