Gene therapy meets CRISPR

gene therapyThe 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 CRISPR diagnostics era is here

ZikaTest_Paper-based-results-002-1024x576Elementary dear Watson, we should have expected that. The CRISPR wave is hitting diagnostics, with a new high sensitivity detection platform named after Arthur Conan Doyle’s popular detective. The acronym SHERLOCK stands for “Specific High Sensitivity Enzymatic Reporter UnLOCKing”. While the technique is used in thousands of labs to turn genes on and off, CRISPR embarks also on epidemiology and learns how to identify nucleic acids from viral and bacterial pathogens to diagnose infections. The paper published in Science by James Collins, Feng Zhang and colleagues heralds a new generation of low cost diagnostic tests with single-base specificity, easy to use even when oubreaks occur in remote areas. Continue reading

Editing cancer at IEO

pelicci quadrotto

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

Targeting Huntington at CattaneoLab

huntingtonCRISPR 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.

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