Blood stem cells getting ready for editing

editing genomicoDo you remember the alarm about the cancer risks of CRISPR? According to a study published in Cell Stem Cell by Italian researchers, the DNA-damage response is not such a big problem in edited cells. At least not in hematopoietic stem cells edited with highly specific nucleases. Continue reading

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