FDA approves first test of CRISPR for genetic defect causing sickle cell disease

Re:FDA approves first test of CRISPR for genetic defect caus... by refurb on Wednesday April 07, 2021 @10:45PM (#26734527)

Sickle cell anemia can be "cured" by inducing expression of fetal hemoglobin (fHb). You don't need to shut off the defective gene and the disease is effectively "cured" if you replace enough scHb with fHb.

So you remove bone marrow from the patient, modify the cells, put them back in. The modified cells produce fHb at a high enough level that symptoms of Sickle Cell go away.

Not that different than the already approved CAR-T therapies (with the exception they don't use CRISPR for the genetic modification).

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Re:FDA approves first test of CRISPR for genetic defect caus... by maxerickson on Wednesday April 07, 2021 @10:51PM (#26734563)

This treatment modifies the adult hemoglobin gene.

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Re:FDA approves first test of CRISPR for genetic defect caus... by refurb on Thursday April 08, 2021 @10:28PM (#26746092)

That's what I get for not reading the article. But the point stands on correcting genetics for hematopoetic cells. It's possible to fix a small number of cells, then reimplant (often depleting the cells in the person first) and they will replicate.

Easier than trying to fix the cells in a solid organ.

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