Another CRISPR step in the way out of congenital muscular dystrophy type 1A (MDC1A) is announced by Ronald Cohn and colleagues in Nature this week. This is still preclinical research in mice, but the indirect approach presented by the Canadian team holds great promise.Continue reading
Researchers from Penn Medicine and Children’s Hospital of Philadelphia have fixed a lethal mutation in the prenatal mouse models of a rare pulmonary disease. The hope is that the approach of in utero editing described in Science Translational Medicine will work for other congenital lung diseases as well.
I asked one of the corresponding authors, Edward Morrison, scientific director of the Penn’s Institute for Regenerative Medicine, to explain what they have done and what to expect next. See Q&A below. Continue reading
If you know the enemy and know yourself, you need not fear the result of a hundred battles. The military strategist Sun Tzu wrote it over two thousand years ago, but this quote could also apply to oncology research in the CRISPR era. Identifying the weak points of cancer cells is the first step to hit new molecular targets with the next generation of drugs.
The good news is that the Wellcome Sanger Institute has taken a giant leap toward this goal, drawing up a list of 600 candidate genes. The study just published in Nature by Mathew Garnett’s team comes with a twin paper by the Broad Institute, confirming the results by following an alternative approach. In a four-year tour de force of functional genomics, Sanger’s researchers used CRISPR to disrupt every gene in over 300 cancer models from 30 cancer types. From this amount of data, they developed a prioritization system which will guide big pharma’s hunt for new drugs.
It’s another CRISPR first: fixing a hereditary disease in utero. Scientists from Pennsylvania University and the Children’s Hospital of Philadelphia used a base editor to rescue tyrosinemia in fetal mice. “The results of this proof-of-concept work demonstrate the possibility of efficiently performing gene editing before birth, pointing to a potential new therapeutic approach for selected congenital disorders,” Avery C. Rossidis and colleagues write in Nature Medicine. Continue reading
Interview given to Anna Meldolesi (Corriere della sera, 15 May 2018)
The CRISPR biomedical duel between China and the US has been called “Sputnik 2.0”. Is Europe being left behind?
JD: As with any disruptive technology, there is intense competition to lead. However, unlike the space race, the CRISPR research effort is global and more collaborative. We consistently see key advances in CRISPR technology shared through scientific papers, written and read by research teams around the world. This collective approach has helped to democratize the technology. However, differing regulations across countries may impact how we ultimately translate research into real-world applications that can benefit the most number of people with the most need. Researchers in Europe have made valuable contributions to the development and application of CRISPR and will continue to play a role in establishing global standards. Continue reading
The first patient edited “in vivo” last week is a breaking news story, and zinc finger nuclease ZFN must be credited for the accomplishment. A putatively outdated system stealing the scene from the most celebrated technique for gene editing is a bit like Carl Lewis beating Usain Bolt at the Rio Olympics. Any wonder that tweets by some biotech-enthusiasts had something of a derby atmosphere, while many inattentive readers thought it was CRISPR stuff, as lay people never heard of ZFN before. Continue reading