First sickle cell patient treated with #CRISPR gene-editing still thriving ▻https://www.npr.org/sections/health-shots/2021/12/31/1067400512/first-sickle-cell-patient-treated-with-crispr-gene-editing-still-thriving
She’s doing so well for so long that she’s officially no longer in the landmark study ▻https://clinicaltrials.gov/ct2/show/NCT03745287?term=crispr&cond=sickle+cell&draw=2&rank=] she volunteered for. That involved doctors taking cells out of her bone marrow, and editing a gene in the cells in their lab, using the revolutionary gene-editing technique known as CRISPR.
CRISPR allows scientists to make very precise changes in DNA much more easily than ever before. Many think it will revolutionize medicine.
Doctors then infused billions of the modified cells back into Gray’s body. The hope was the edited cells would produce a protein known as fetal hemoglobin, alleviating the symptoms of sickle cell.
And it appears to have worked, for Gray and other patients. Doctors have now treated at least 45 patients with sickle cell and a related condition known as beta thalassemia, and reported data indicating it’s working for at least 22 of them.