Innovative Gene Therapy Treats Deafness

Incorporating genetic material into the cells of the inner ear helps innovatively treat deafness, as per a study from Tel Aviv University (TAU), published in the journal EMBO Molecular Medicine. According to the World Health Organization, there are about half a billion people with hearing loss around the world today, and this figure is expected to double in the coming decades. One in every 200 children is born with a hearing impairment, and one in every 1,000 is born deaf. In about half of these cases, deafness is caused by a genetic mutation. There are currently about 100 different genes associated with hereditary deafness, making it a common sensory disability worldwide.

Gene therapy in hearing loss

"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. 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", says Professor Karen Avraham, Department of Human Molecular Genetics and Biochemistry at TAU's Sackler Faculty of Medicine and Sagol School of Neuroscience.

The team utilized gene therapy technology where a genetic material "replaces" the genetic defect – mutated gene and enables the cells to mature and function normally.

This prevented the gradual deterioration of hearing in mice models that had a genetic mutation for deafness, and received treatment soon after birth. The mice's hearing was monitored using both physiological and behavioral tests and complete restoration of normal hearing and sensitivity was observed.

"The magnitude of hearing recovery is impressive. This study is a part of a growing body of literature showing that gene therapy can be successfully applied to mouse models of hereditary hearing loss, and it illustrates the enormous potential of gene therapy as a treatment for deafness", says Prof. Wade Chien, MD, from the NIDCD/NIH Inner Ear Gene Therapy Program and Johns Hopkins School of Medicine.

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The study also stirs up the development of similar therapies for other mutations that cause deafness.

Source-Medindia