So far we have learned that CRISPR may turn a faulty gene off by cutting and mutating its sequence. But what if we want to proceed more cautiously and avoid permanent changes to the genome? We could leave the target gene intact but ineffective, by intercepting and destroying the RNA messages with which it gives the wrong orders to the diseased cells. In this way it would be easier to go back if necessary. The good news is that CRISPR is a jack-of-all-trades, well-suited for the task, and the new approach (call it RNA targeting with CRISPR) is going to help to study human biology and diseases. One of the technique pioneer, Feng Zhang, has demonstrated in Nature last week that it can efficiently target RNA in mammalian cells (and also plants), equalizing and even surpassing the performance of the current tool of choice for RNA knockdown (RNA interference). In short, besides advancing its career as DNA editor, CRISPR has also found a second job in the RNA business. Continue reading
The University of Berkeley has opened a glimpse into the way bacteria use CRISPR, the microbial immune system that inspired the invention of the method for genetic modification also known as CRISPR. The paper published in Science by Jennifer Doudna’s team is a fascinating piece of basic research and scientists are hopeful they will be able to turn the discovery into a new biotech tool. Continue reading
The Italian city of Terni is now a spot on the map of cutting-edge research due to its new genetic-ecology lab, which is getting involved in the Target Malaria project funded by the Bill & Melinda Gates Foundation. For a couple of days, citizens are allowed to visit the facility which is part of the Genomics, Genetics and Biology Innovation Pole. That’s an example of real public engagement: everybody can talk to researchers and watch videos, but also enter the climatic rooms simulating tropical conditions and see the cages for the insects which are the tiny heroes and the target of a daring scientific challenge. The mission here in Terni indeed is to investigate if the idea of controlling genetically malaria, by introducing self-destroying Anopheles gambiae into wild mosquitoes populations, is set to work in real world situations. Continue reading
The aim is engaging: to treat an increasing number of diseases by correcting the underlying genetic defects. And researchers are breathing optimism at last. The San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) in Milan has already treated 58 patients (including ADA-SCID, leukodystrophy, Wiskott-Aldrich syndrome and beta-thalassemia) and the count is approaching 300 worldwide. Moreover the promise of genome editing is looming on the horizon. We discussed the present and future of the field with the SR-Tiget director Luigi Naldini, who contributed to the latest report on human genome editing published by the US National Academies of Sciences and Medicine. Continue reading
The second leg of the journey among leading labs takes us to the European Institute of Oncology in Milan. CRISPR’s potential is particularly exciting for oncology, as tumors are caused by multiple mutations and the new technique of genome editing is multiplexable, meaning it may target several genes at the same time. IEO scientific co-director Pier Giuseppe Pelicci has shared his enthusiasm with us.
“In our lab we are using CRISPR in 3 broad research areas. In the first area we follow the classic way, by disrupting the genes we want to study in order to understand their functions. CRISPR can do it much better than the previous techniques. It’s fast, very cheap and easy to handle. Before CRISPR we could carry out an experiment every 6 months, after CRISPR we can do one every week. It’s like altering the flow of time.” Continue reading
CRISPR is radically changing the way researchers work, by allowing better, faster, and cheaper experiments. This blog will tell, among other things, how leading labs are using the most popular technique for genome editing. Let the dance begin with the Laboratory of Stem Cell Biology and Pharmacology of Neurodegenerative Diseases of the University of Milan (CattaneoLab). The group directed by Elena Cattaneo is busy unveiling the molecular basis of neurodegeneration in Huntington’s disease with the help of CRISPR, as pharmacologist Chiara Zuccato explains.