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.
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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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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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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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.
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!
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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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.
As we become increasingly able to reshape genomes, the need to organize information in efficient and accessible ways continues to grow. More than a thousand plant genomes have now been sequenced, with the pace accelerating, so it is good news that the plant genetics community can rely on PubPlant, a new interactive, updatable, and freely accessible atlas introduced in Frontiers in Plant Science. The magazine The Scientist has written about it, likening PubPlant to a kind of Google Maps for plant DNA, designed to help researchers more quickly pinpoint key genomic regions linked to traits such as disease resistance, nutritional quality, or climate adaptation. (Image Credit: Salk Institute-USDA)
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