Gene Therapy: A Threat To The Deaf Community?

Typically, gene therapy involves combining a therapeutic gene with a vehicle known as a viral vector. This vector is used to deliver the therapeutic gene into a target cell by a process known as transduction. In the case of the inner ear, there is a low transduction efficiency in sensory cells using such viral vectors, including the vector known as AAV1. As a result, there has been variable and inefficient uptake of therapeutic genes.

A recent study in mice, however, published in the journal Molecular Therapy, describes a new method for delivering genes to the sensory hair cells of the inner ear as a potential treatment for deafness. This research describes a new type of viral vector, exo-AAV1, which is more efficient than AAV1 and which may be an effective viral vector for delivering therapeutic genes to treat hereditary deafness by gene therapy.

The use of exosome-associated viruses raises important questions about risks (and unwanted side-effects). There is, for example, the risk of transferring genes that might facilitate the spread of disease through the delivery of genetic material and/or pathogenic...