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Stem cell gene-editing method may be breakthrough for sickle cell research

Researchers have cured mice with a genetic blood disease by correcting mutated genes in blood cell-producing stem cells. The method offers a new therapeutic approach for treating blood disorders, such as sickle cell disease and thalassemia.

The gene-editing technique can be administered easily into living animals and notably decreases off-target effects (side effects), unlike the CRISPR technique, which displays problems in those regards.

The study, “In vivo correction of anaemia in β-thalassemic mice by γPNA-mediated gene editing with nanoparticle delivery,” was published in Nature Communications.