Shanghai (Xinhua News) – A four-year-old Pakistani girl, identified as Elsa, has successfully recovered from severe beta-thalassemia after receiving groundbreaking gene therapy at Fudan University’s Children’s Hospital in Shanghai. After over four months of treatment and observation, Elsa no longer requires regular blood transfusions and is returning to a normal life.
Elsa arrived in Shanghai on January 8 and became the first foreign child — and the youngest — to undergo China’s original gene-editing treatment for thalassemia, a hereditary blood disorder. Thalassemia is common in the Mediterranean, the Middle East, Africa, and Southeast Asia.
Elsa’s father, Muhammad Adeel, said she is the second of three daughters. Although their eldest child was healthy, the couple later discovered they were both carriers of the disorder, which left Elsa dependent on frequent transfusions to survive.
After learning about advanced treatment options online, Adeel contacted Chinese researchers and brought Elsa to Shanghai. At Fudan University’s Children’s Hospital, doctors collected her stem cells and used a highly precise Transformer Base Editor (TBE)—developed by ShanghaiTech University—to edit the faulty genes. The edited cells were then reintroduced into her body, enabling her to produce healthy hemoglobin.
Professor Zhai Xiaowen, vice president of the hospital, explained that using the patient’s own stem cells eliminates the need for a donor, avoiding transplant rejection risks. Elsa’s hemoglobin levels have now normalized, and she can lead a healthy life and attend school.
On May 20, the hospital held a farewell ceremony for Elsa, with her parents expressing deep gratitude. Adeel, a fiber-optic sensing scientist, called the experience a “moment of pride” and praised the medical team for their life-saving work.
