Gene Editing Used to Successfully Treat Thalassemia in China
What’s new: Doctors in China have used gene editing to treat patients with thalassemia, the first time the technique has been successfully implemented in the country.
Medical scientists at Central South University’s Xiangya Hospital in Central China’s Hunan province said those receiving the treatment had gone 87 days without blood transfusions, a common remedy for the hereditary blood disorder.
Experts told Caixin that the technique still carried a certain amount of clinical risk and further experimentation in China many be restricted by policy shortcomings and ethical concerns.
The background: Thalassemia is a genetic condition that causes the body to produce lower-than-normal amounts of hemoglobin, the protein in red blood cells that carries oxygen. Symptoms include anemia, tiredness and pale or yellowish skin.
While people with mild thalassemia rarely require follow-up treatment, those with severe forms of the disorder may need to undergo frequent blood transfusions and take steps to control their iron levels.
While gene therapy has been approved in some countries for certain types of thalassemia, China is yet to OK the practice.
Around 8% of people in Hunan carry the gene that causes thalassemia, the provincial health commission said in May. Hunan and the island province of Hainan have the highest prevalence of thalassemia in China.
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