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  • Writer's pictureLaurent DECORY

CRISPR treatment used on patient for the first time

Updated: Mar 24, 2020

Patient with congenital blindness tested with CRISPR treatment for the first time.

#genomics #patientoutcomes


CRISPR technology, often referred to as ‘molecular scissors,’ is used to edit the DNA, for example aiming to remove a mutated DNA section and replacing it by non mutated one. There are however questions if CRISPR could inadvertently cut other parts of the DNA as well.

Until now, CRISPR-engineered treatments such as CAR-T were created by removing cells from the patients, editing them and then re-injected.

In this world-first, a patient with congenital blindness was tested in-vivo with CRISPR treatment for the first time, as part of a Phase I/II trial conducted by Editas and Allergan. Doctors inject drops of a fluid containing the CRISPR-based treatment beneath the retina, where it deletes the mutation and hopefully will restore. vision

If successful, 18 total patients are to be treated in this Phase I/II trial.

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