Novel gene therapy significantly reduces hearing loss linked to rare disease in mouse model

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Novel gene therapy significantly reduces hearing loss linked to rare disease in mouse model
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An international team led by researchers at UCL and the NIHR Great Ormond Street Hospital Biomedical Research Centre have developed in mice a gene therapy that significantly reduces the hearing loss associated with Norrie disease.

loss. This research is aimed at slowing or preventing the hearing loss and thus the dual sensory deprivation that severely restricts the lives of those with Norrie disease., which are only partly effective in Norrie disease. This research in mice, published incan be used to prevent the death of the vitally important hair cells in the cochlea—the snail shell-like part of the inner ear responsible for sensing sound—and stop the loss of hearing associated with Norrie disease.

A slice through a cochlea after gene therapy shows replaced norrin protein in pink / magenta. Credit: Professor Jane Sowden et al

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