Engineering lymphocytes to recognize cancer cells is a strategy that has already produced convincing clinical results thanks to CAR-T therapy. But this is not theonly approach on the horizon. An emerging alternative is TCR-engineered lymphocytes, where TCR stands for T-cell receptors.
Here you can read a selection of notable comments about the landmark paper on in vivo genome-editing published in the New England Journal of Medicineon 26 June. The trial, conducted in the UK and New Zealand, produced the first-ever clinical data supporting the safety and efficacy of intravenous infusion of a single-dose CRISPR treatment. The treatment, developed by two US-based companies (Intellia Therapeutics and Regeneron Pharmaceuticals) targets a rare and fatal condition called transthyretin amyloidosis.
Jennifer Doudna (CRISPR co-inventor and co-founder of Intellia): “It’s a critical first step in being able to inactivate, repair, or replace any gene that causes disease, anywhere in the body” (source Science).