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CRISPR used to treat rare genetic disease in promising phase 1 trial

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Scientists are reporting the first clinical data showing that CRISPR gene editing can be done safely and effectively inside the body. CRISPR was injected directly into the bloodstream of patients with a rare genetic disease, and appeared to work better than current treatments with no serious side effects.

The CRISPR gene-editing tool, which makes precise cut-and-paste edits to the genome inside cells, has shown tremendous promise in treating a wide range of diseases. It’s currently the subject of many human trials, but in almost all of them the actual editing takes place outside the body – cells are removed from the patient, edited and then returned to the body. Another recent study involved injections of CRISPR into the eye to correct a genetic form of blindness, but the results have yet to be published.

Now researchers have released interim data of the first six patients in an ongoing Phase 1 trial conducted by Intellia Therapeutics and Regeneron Pharmaceuticals, marking the first published results of a CRISPR clinical trial involving human gene editing in vivo.

The trial is investigating a CRISPR candidate called NTLA-2001 as a treatment for a disease called transthyretin amyloidosis (ATTR amyloidosis). This rare hereditary disease occurs due to mutations in the TTR gene, which causes a patient to produce misfolded transthyretin (TTR) proteins. These abnormal proteins then build up on nerves and around organs, leading to pain and complications, and can be fatal.

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