New gene therapy study addresses treatment methods for deafness.
"In this study we focused on genetic deafness caused by a mutation in the gene SYNE4 -- a rare deafness discovered by our lab several years ago in two Israeli families, and since then identified in Turkey and the UK as well," Professor Avraham reports. "Children inheriting the defective gene from both parents are born with normal hearing, but they gradually lose their hearing during childhood. The mutation causes mislocalization of cell nuclei in the hair cells inside the cochlea of the inner ear, which serve as soundwave receptors and are essential for hearing. This defect leads to the degeneration and eventual death of hair cells."
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"We implemented an innovative gene therapy technology: we created a harmless synthetic virus and used it to deliver genetic material -- a normal version of the gene that is defective in both the mouse model and the affected human families," says Shahar Taiber, one of Professor Avraham's students on the combined MD-PhD track. "We injected the virus into the inner ear of the mice, so that it entered the hair cells and released its genetic payload. By so doing, we repaired the defect in the hair cells and enabled them to mature and function normally."
Source: Science Daily