CRISPR/Cas9 technology could be used as soon as 12-18 months in approved experimental trials to treat sickle cell disease and severe combined immunodeficiency.
Matthew Porteus, MD, PhD, of the Institute of Stem Cell Biology and Regenerative Medicine at Stanford (Calif.) University, gave the projected timeline during a U.S. Senate Committee on Health, Education, Labor & Pensions hearing on gene editing technology held on Nov. 14.
"The CRISPR/Cas9 system allows scientists to correct disease-causing mutations in human cells with unprecedented efficiencies. In my lab, for example, we can correct the mutation that causes sickle cell disease in patient derived blood stem cells at a frequency of 50-80%. For severe combined immunodeficiency (“bubble boy disease”) our correction frequency is 40-50%. For both (disorders) the correction is highly specific and exceeds the level of correction by 5-10 fold over the efficiency that is predicted to be needed to cure a patient. We have been working closely with the FDA (Food and Drug Administration) to bring these therapies to patients in the next 12-18 months," Dr. Porteus said in his prepared statement https://www.help.senate.gov/imo/media/doc/Porteus.pdf.
The entire hearing can be viewed at https://www.help.senate.gov/hearings/gene-editing-technology-innovation-and-impact.