First U.S. trial uses non-viral CRISPR to correct sickle cell mutation
Lucian Nemoiu
University of California San Francisco Medical Center Nov 26 2024 UCSF Benioff Children's Hospital Oakland is enrolling patients in an innovative clinical trial that seeks to cure sickle cell disease. The trial is the first in the U.S. to apply non-viral CRISPR-Cas9 gene-editing technology in humans to directly correct the genetic mutation that causes the disease. The research involves taking the patient's blood stem cells to correct the mutation and returning those edited cells to the patient
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