And though comprehensive sickle cell care — at dedicated centers with expert hematologists, social workers and pain management specialists — reduces hospitalizations, and is the standard for diseases like cystic fibrosis and hemophilia, which do not disproportionately affect Black people, these centers are few and far between for sickle cell.
Into this complicated landscape enters the possibility of gene therapy.
It’s important to note that this isn’t the first cure for sickle cell.
is expected to review another gene therapy from the company Bluebird Bio that targets sickle cell disease but does not use CRISPR; this was the therapy Mr. Holmes received as part of the N.I.H.
When she was 17 and hospitalized, facing the reality of her chronic illness, she told her mother that she was ready to pursue gene therapy.
Persons:
Holmes, Elizabeth Ford, Ford
Organizations:
Vertex Pharmaceuticals, CRISPR Therapeutics, Bluebird