The genome-editing method involving CRISPR and Cas9 has been called into duty for a wide variety of jobs, from cutting integrated HIV out of the human genome to turning off genes in primates. In a new development published in Genome Research, researchers have used CRISPR/Cas9 in human cell lines to rewrite a mutant gene that causes a blood disorder called β-thalassemia.
“It is an incremental step forward to use genome editing to correct disease-causing mutations,” said Paul Schmidt from Children’s Hospital Boston and Harvard Medical School who did not participate in the study. Still, he added, there are a number of hurdles that “need to be overcome before it can be used in the clinic.”
β-thalassemia is caused by a mutation in the HBB gene, resulting in a severe hemoglobin deficiency. It’s estimated to affect one in 100,000 people globally, including one in 10,000 in Europe. Patients require transfusions that can overload them with iron, a complication that also requires treatment. Some researchers are working to develop a gene therapy for the problematic gene, but so far there is no cure.
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