Several dozen patients with sickle cell disease will be among the first in the US treated for a genetic disease with the experimental gene-editing technology CRISPR
"This is an extraordinary time" in the treatment of sickle cell disease, said Dr. Alexis Thompson, hematology section head at the Ann and Robert H. Lurie Children's Hospital of Chicago, who is not involved in the trial."It's terribly exciting that there is so much attention on cutting-edge science and a condition that has lagged far behind many other medically important diseases.
"We are going to need, at some point, to be able to measure these to say which ones of these was better," Thompson said,"and are there subsets of patients for which one of these is superior?"Experts say it's too early to tell how the CRISPR patients will fare long-term -- what sort of improvement researchers see versus the risks they bear with gene-editing technology and chemotherapy.
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