Teresa Blankmeyer Burke considers the problematic nature of gene therapy research aimed at eliminating hereditary deafness.
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 proteins. These risks, while important, are not as pressing, however, as the larger issue of whether researchers should conduct research that threatens to eradicate a community.
Members of the signing Deaf community argue that research which aims to eliminate or cure deafness is a form of cultural genocide. The argument goes like this: the use of gene therapy to cure hereditary deafness would result in smaller numbers of deaf children.
The views, opinions and positions expressed by these authors and blogs are theirs and do not necessarily represent that of the Bioethics Research Library and Kennedy Institute of Ethics or Georgetown University.