Watson & Crick for the double helix. Doudna & Charpentier for CRISPR. Karikó & Weissman for RNA vaccines… Do two people think better than one or even many? Itai Yanai and Martin J. Lercher suggest so in Nature Biotechnology.
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It’s been about a year since the first CRISPR-based treatment was approved in the United States and Europe. However, those expecting a surge in approvals of new gene-editing therapies may be disappointed. Next in line will likely be another approach to treating sickle cell disease, followed by therapies for TTR amyloidosis and hereditary angioedema around 2026-27. According to The CRISPR Journal, that’s all we can expect over the next 3-5 years. Is Casgevy destined to stand out like a cathedral in the desert? We have a super-versatile platform capable of fixing a myriad of genetic defects, so why is the CRISPR revolution slowing down? To understand the looming crisis and the countermeasures needed, don’t miss Fyodor Urnov’s in-depth editorial entitled “Give Cas a Chance: An Actionable Path to a Platform for CRISPR Cures.”
This photo shows the first American “non-experimental” patient leaving the hospital after completing the CRISPR-based treatment for sickle cell anemia (Casgevy). The New York Times detailed this “official first,” which followed the success of a clinical trial involving dozens of patients like Victoria Gray. We still know little about the first person who is beginning treatment in Europe since this therapy became an “approved drug”. According to Osservatorio Terapie Avanzate he is a young adult (23 years), who arrived in Italy in 2014 and living in the Umbria region, where is being also treated. Undergoing cell extraction and reinfusion of edited cells is an invasive and exhausting process, but now the American Kendric Cromer (12 years old) and other “first patients” can hope to lead full lives—without painful crises or blood transfusions. Best of luck!
Emmanuelle Charpentier and Jennifer Doudna won the Nobel Prize but not the intellectual property dispute and are now ready to give up two patents granted in Europe. The scoop once again comes from Antonio Regalado, who gained fame a few years ago for breaking the CRISPR babies scandal.
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MicroRNAs won Victor Ambros and Gary Ruvkun the 2024 Nobel Prize in Medicine. Thomas Cech (Nobel Laureate for the discovery of catalytic RNA) has found a fun way to explain how they work. His book, which I reviewed a few weeks ago, is a mine of insights and information. Here is a small excerpt.
Continue readingSammy Basso, 28, the longest-living person with progeria and a brilliant mind, has passed away. He was a biologist passionate about genome editing, and we’d like to remember him through this dialogue with David Liu.
Testing of Chardonnay edited to resist downy mildew starts today near Verona, while the prosecco variety awaits its turn in the greenhouse
Forget Odin, the controversial kit that was being sold online by controversial “biohacker” Josiah Zayner (getting people to play around with developing antibiotic-resistant bacteria is certainly not a good idea). At Stanford University they have developed a CRISPRkit for cell-free in vitro experiments that is easy and safe because the target is a harmless pigment. And the great thing is that it costs less than an American coffee (two dollars).
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RNA interference, mRNA vaccines, RNA-guided editing. These are just some of the fields of biomedical research that have exploded in recent years and brought RNA out of the projected shadow of the most famous nucleic acid: DNA. Credit for the ongoing scientific and technological revolution goes to researchers such as Thomas Cech, who have been able to look beyond the double helix and, in many cases, have earned Nobel prizes (the latest being Katalin Karikό for the anti-Covid vaccine). But we also owe a nice gratitude to the strange critters who first exhibited some unexpected phenomena that later became of universal interest.
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Four intense days of talks and discussions on the present and future of agricultural genetics. We will be there at the round table “Social impact of plant biotechnologies: challenges and opportunities” (12 September afternoon). See the full program here.
CRISPeR FRENZY 