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Gaucher's Neurodegenerative Disease can be prevented by prenatal gene therapy, performed in the uterus, according to a first mouse study published today online in Nature Medicine.
The state of Gaucher (GD) is an inherited and irreversible disease that constitutes a metabolic disorder due to the absence of glucocerebrosidase and which causes inflammation of the liver, spleen, bone fragility, anemia and fatigue.
The team of scientists discovered that the application of a viral vector – a modified virus used to introduce genetic material into the nucleus of a cell – into the brain of the fetus of a mouse with GD, allowed the animal to survive for longer and show less brain degeneration, thanks to the modification of one of the deficient enzymes with the disease.
The head of research, Simon Wa Ddington, of University College London, explained that "mice who received the injection in utero were living up to at least 18 weeks after delivery ", a number much higher than the" 15 days "that lasted those rodents who did not
" They showed no symptoms of neurodegeneration, they were fertile and completely mobile ", a- he says about animals having undergone gene therapy, with which they have reconstituted the neuronal expression of glucocerebrosidase. They can be treated after birth with enzymatic substitution therapies, but neurodegeneration is refractory and, in most cases, fatal, so therapies should be started as soon as possible.
Scientists have also developed a method to guide the transfer of vectors into larger brains in the uterus of nonhuman primates by ultrasound, since macaques and humans have very similar neurological systems.
The next steps in the investigation require to determine how the vector evolves throughout the life of the animal, particularly in the central nervous system, and to increase the degree of accuracy of the diagnosis. gene in the uterus. EFE
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