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Gene therapy prevented deafness in mice with genetic defects

Scientists to use this approach & treat deafness due to other mutations also

04-Jan-2021

Key points from article :

Novel treatment prevented deterioration of hearing in mice with genetic defects.

Delivered healthy genetic material into the inner ear cells of these mice.

“Focused on genetic deafness caused by a mutation in gene SYNE4” - Prof Avraham, TAU.

“Children inheriting the defective gene are born with normal but gradually lose their hearing"

"Mutation leads to the degeneration and eventual death of hair cells".

Harmless synthetic virus delivered normal version of the gene.

Injected the virus into the inner ear of the mice - repaired defects in hair cells of mice.

Treatment was administered soon after birth.

Treated mice developed normal hearing, with sensitivity almost identical to that of healthy mice.

Research by Tel Aviv University (TAU) published in EMBO Molecular Medicine.

Mentioned in this article:

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EMBO Molecular Medicine

Scientific journal providing information from the field of molecular medicine.

Karen Avraham

Vice Dean, Faculty of Medicine, Tel Aviv University

Tel Aviv University

Public Research university.

Topics mentioned on this page:
Gene Therapy