Here is what we have been writingCRISPR gene therapy trials demonstrate safety Two similar CRISPR gene therapy studies were published in high impact peer-reviewed journals. Both studies investigate CRISPR-Cas9-edited T cells as a treatment for cancer, and both studies report the gene therapy to be feasible and safe. Now, the stage is set for the next significant advances in cancer treatment. Reversing diabetes with CRISPR and patient stem cells A rare genetic form of diabetes has been reversed in mice. Researchers use CRISPR-Cas9 gene editing to correct a diabetes-causing mutation in stem cells derived from patients. Then they show the edited stem cells can reverse severe diabetes in mice. Breaking the PAM Barrier In an exciting research breakthrough, Ben Kleinstiver and colleagues at Harvard have engineered novel Cas9 variants that can target the entire human genome. It paves the way for research and clinical trials using genome editing in sites that used to be completely inaccessible. CRISPR as a bacteria killer In a world's first, Locus Biosciences has enrolled the first patients in a clinical trial using bacteriophage boosted with CRISPR to cure urinary tract infections. The completely new antibacterial principle for curing disease comes as multi-drug resistance is on the rise. Curing cystic fibrosis with CRISPR, stem cells and mini-organs Dutch scientist Hans Clevers and colleagues from Utrecht have used CRISPR base editors to cure cystic fibrosis in mini-organs derived from patient stem cells. Encouragingly no off-target effects were found, and the gene therapy may be safe to use for repairing damaged tissue. |