Regulators in the UK have given CRISPR Therapeutics the green light to conduct a clinical trial of its gene-editing based therapy for beta thalassemia, the Telegraph reports.
For the trial, the Telegraph says CRISPR Therapeutics researchers will be removing hematopoietic stem cells from patients and editing them in the lab to produce fetal hemoglobin, whose expression is typically repressed in adults through the BCL11A gene. By ramping up its production in adults with beta thalassemia, the firm hopes to boost the amount of oxygen carried to cells to treat the disease.
"Certainly, 2018 promises to be the big year for clinical trials using CRISPR-based genome editing," University College London's Helen O'Neill tells the Telegraph.
In the US, Editas Medicine has said it plans to soon ask the Food and Drug Administration to conduct a clinical trial of its gene-editing therapy for congenital blindness, and researchers at the University of Pennsylvania have also been recruiting patients to its clinical trial testing the use of CRISPR/Cas9 to edit two genes to bolster cancer patients' immune systems. Similar trials have already begun in China.