A new medical study published online by the journal Nature Medicine on the 16th shows that gene therapy can prevent early-onset fatal neurodegenerative disease, Gaucher disease. A team of British scientists delivered a viral vector to the central nervous system of the fetus, which was engineered to re-express a specific coding gene to alleviate brain degeneration in the fetus.
Gaucher disease is a genetic disease. Common symptoms include hepatosplenomegaly, bone fragility, bone pain, anemia, fatigue, and vulnerability to contusion. These symptoms are caused by the body's inability to properly break down lipids and cause them to accumulate. Some mild Gaucher disease can be treated by enzyme replacement therapy after delivery. However, severe Gaucher disease is currently incurable, which causes irreversible early onset neurological regression and is usually fatal. This prognosis means that treatment must be initiated as soon as possible.
Gene therapy refers to the introduction of a foreign normal gene into a target cell to correct or compensate for diseases caused by genetic defects and abnormalities for therapeutic purposes. This time, University of London researcher Simon Waddington and colleagues delivered a viral vector to the central nervous system of the fetus through surgery. This engineered viral vector re-expresses a specific coding gene that encodes an enzyme that is lacking in patients with Gaucher disease. After the treatment of mice in the uterus, brain degeneration was relieved and the survival period was significantly longer than that of untreated mice.
To achieve clinical application, the research team also sought to use ultrasound to direct the delivery of similar viral vectors into the brains of unborn non-human primates.
The researchers said that further research is needed in the future to determine the extent to which viral vectors need to continue to be expressed during the life cycle of the target animal, especially in the larger neurological system of non-human primates. In addition, such gene therapy also requires early and accurate diagnosis of prenatal disease in the subject. (Reporter Zhang Mengran)
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