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# FDA Approves CRISPR Trial to Correct Sickle Cell Gene Defect
- URL: https://www.fdaweb.com/fda-approves-crispr-trial-to-correct-sickle-cell-gene-defect/
- Published: 2021-03-30T12:00:00.000Z
- Updated: 2026-09-14T16:52:47.000Z
- Author: David McFarland
- Tags: Biologics, #legacy-id-D5148995

FDA has approved for clinical trials an experimental treatment using a CRISPR-based therapy to directly correct the mutation in the beta-globin gene responsible for sickle cell disease. The technique was developed at the Innovative Genome Institute, a joint research collaboration of the University of California, Berkeley, and the University of California, San Francisco (UCSF). A [statement](https://news.berkeley.edu/2021/03/30/fda-approves-first-test-of-crispr-to-correct-genetic-defect-causing-sickle-cell-disease/?ref=fdaweb.com) says the trials are expected to last four years and will be led by doctors at UCSF’s Benioff Children’s Hospital Oakland and UCLA’s Broad Stem Cell Research Center. The statement says the doctors plan to begin this summer to enroll six adults and three adolescents with severe sickle cell disease.

In the trial, researchers will use CRISPR-Cas9 to replace the defective beta-globin gene with a repaired version to create normal, adult red blood cells and cure the disorder.