Prenatal gene therapy can prevent fatal neurodegeneration diseases

recently,
gene therapy could prevent early-on-oncr fatal neurodegenerative diseases in mice, according to a paper published online today.
is a genetic disorder with common symptoms including enlarged liver and spleen, fragile bones, bone pain, anemia, fatigue and vulnerability to bruising. These symptoms stem from the body's inability to break down lipids properly, causing them to build up. Some mild Goshe disease can be treated with postparto enzyme replacement therapy, but severe goshe disease, which causes irreversible early neurological degeneration, is currently incurable and often fatal. This prognosmation means that treatment must be started as early as possible.
Simon Waddington and colleagues at University College London used surgery to deliver a viral vector to the central nervous system of a fetal rat. The viral vector has been modified to re-express a special coding gene, an enzyme lacking in patients with Goshe disease. After receiving this treatment, the degeneration of the brain was alleviated and the survival period was significantly longer than that of the untreated mice. For clinical use, the researchers also managed to use ultrasound to direct similar viral gene transfer vectors to the larger brains of unborn non-human primates.
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 neural central systems of non-human primates. In addition, this type of gene therapy requires early and accurate diagnosis of prenatal diseases. (Source: China Science Daily Tang 1 Dust/Compilation)