Health
New gene therapy delivery system restores hearing in mice
Gene therapy can treat hereditary hearing loss in newborn mice. However, treatment in adult animals is more challenging due to the location of the cochlea and the risk of damage to inner ear structures. A new study by an international team of researchers has now demonstrated a new method of delivering drugs to the inner ear. Researchers have successfully restored hearing in deaf mice using a gene therapy that harnesses the natural flow of cerebrospinal fluid and cochlear aqueduct to repair inner ear hair cells.
their discovery scientific translational medicine In an article titled “”,Delivery of gene therapy via cerebrospinal fluid conduits to restore hearing in adult mice“
“Inner ear gene therapy recently effectively restored hearing in newborn mice, but this becomes difficult in adulthood because the cochlea embedded within the temporal bone is structurally inaccessible,” the researchers wrote. there is “Alternative routes of delivery could advance hearing research and may also prove useful when applied to humans with progressive hereditary hearing loss. Cerebrospinal fluid flow is emerging as a novel approach for drug delivery throughout the brain in rodents and humans.Cerebral spinal fluid and inner ear fluid are connected through a bony passage called the cochlear aqueduct. However, no previous studies have explored the possibility of cerebrospinal fluid-mediated gene therapy to restore hearing in adult deaf mice. , showed that the mouse cochlear aqueduct exhibits lymphatic-like features.”
“These findings indicate that cerebrospinal fluid transport constitutes a viable pathway for gene delivery to the inner ear in adults, and may be useful for using gene therapy to restore hearing in humans.” It could be an important step forward in the future,” said Professor Miken Nedergaard, MD, DMSc, from the Center. He is a PhD in Translational Neuromedicine at the University of Copenhagen and senior author of a new study.
Although hair cells do not regenerate naturally in humans and other mammals, gene therapy has shown promise, and another study restores hair cell function in neonates and very young mice. have succeeded in However, as both mice and humans age, the cochlea becomes surrounded by the temporal bone. At present, attempts to reach the cochlea and perform surgical gene therapy risk damaging this sensitive area and altering hearing.
In a new study, researchers describe passages within the cochlea called aqueducts. Using a number of imagination and modeling techniques, the researchers were able to develop a detailed depiction of how fluid from other parts of the brain flows through the cochlear aqueduct into the inner ear. . The researchers then injected adeno-associated virus into the cisterna magna (a large reservoir of cerebrospinal fluid) at the base of the skull. The virus entered the inner ear via the cochlear aqueduct and was given gene therapy to express a protein called vesicular glutamate transporter-3. This enabled hair cells to signal and restore hearing in adult deaf mice.
“This new route of delivery to the ear may not only help advance hearing research, but may also prove useful when applied to humans with progressive hereditary hearing loss.” Yes,” said Nedergaard.
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