as mentioned in Because hair cells are also critical for the sense of balance, mice with damaged or missing hair cells show balance abnormalities.
On their quest to restore hearing through gene therapy, scientists have long sought ways to improve gene delivery into hair cells.
Post-treatment tests revealed that the gene entered between 30 and 70 percent of hair cells, reaching both inner and outer hair cells.
The team now plans to improve their gene-delivery technique in an attempt to reach an even greater proportion of hair cells.
Using a novel form of gene therapy, scientists from Harvard Medical School and the Massachusetts General Hospital have managed to restore partial hearing and balance in mice born with a genetic condition that affects both.
As it stated in
New gene delivery therapy restores partial hearing, balance in deaf mice
Rescue of Hearing by Gene Delivery to Inner-Ear Hair Cells Using Exosome-Associated AAV.
Hair cells are the sensory receptors of both the auditory system and the vestibular system in our ears — and the ears of all vertebrates.
Scientists have used AAV for genetic delivery before, but hair cells proved very difficult to penetrate, and this is where the exosomes kick in.
Using an innovative genetic editing technique, researchers have managed to partially restore both heating and balance in mice born with a condition that affects both.
The mice were unable to hear even the loudest of sounds and had visible balance problems.
As it stated in
New gene therapy for Pompe disease may replace currently available treatments
The gene Therapy, demonstrated in mice, is described in a new study published online in the journal Molecular Therapy – Methods & Clinical Development.
Pompe disease is an inherited condition that affects approximately 1 in 20,000 babies and can also appear in adulthood.
The Therapy uses a modified virus to deliver a gene to the liver where it produces GAA, an enzyme missing in people with Pompe disease.
Gene Therapy could help these patients.”
Some infants still die from Pompe disease.
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