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First CRISPR Human Clinical Trial Gets a Green Light from the U.S.

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On June 21, an advisory committee at the US National Institutes of Health approved a proposal to use CRISPR-Cas9 to help augment cancer therapies that rely on enlisting a patient’s T cells, a type of immune cell.

The researchers will remove T cells from 18 patients with melanoma, sarcoma or myeloma, and perform three CRISPR edits on them. The third is defensive: it will remove the gene for a protein that identifies the T cells as immune cells and prevent the cancer cells from disabling them. Editas Biotechnologies in Cambridge, Massachusetts has said that it wants to use CRISPR in a clinical trial for a rare form of blindness as soon as 2017.

CRISPR has courted most attention because of its ease of use, however the T-cell trial will not be the first test of the efficacy of using gene editing to fight diseases.

Last week, researchers at Great Ormond Street Hospital for Children in London began a safety study with 10 children using a similar technique called TALENS. Instead of using a patient’s own cells, the system uses T cells from a donor that have been edited to remove genes that would cause the patient’s body to reject them.

The gene editing then directs the T cells to attack the cancer and protects the cells from other immunotherapy drugs.

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Article originally posted at bit.ly

Post Author: Carla Parsons

1 thought on “First CRISPR Human Clinical Trial Gets a Green Light from the U.S.

    Daniel Black

    (October 29, 2017 - 5:06 am)

    Katy, has your doctor looked at this one?

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