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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.


 

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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