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Personalized Treatment of Genetic Hearing Loss through RNAi

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. Personalized Treatment of Genetic Hearing Loss through RNAi. An estimated 2,700,000 people in the US have autosomal dominant progressive hearing loss. At this time, there is no effective treatment for these persons. The World Health Organization estimates that worldwide 500,000,000 people have at least moderate hearing loss. Last year, these persons spent $5.4 billion on treatment. Broadly speaking, their causes of hearing loss fall into two categories: environmental influences and/or genetic factors. Genetic factors are estimated to account for about 50% of hearing loss and are subdivided into syndromic or non-syndromic types of deafness. Syndromic deafness occurs in association with abnormalities in other parts of the body, while non-syndromic deafness is not associated with other signs and symptoms. Non-syndromic deafness accounts for about 80% of genetic hearing loss and can be diagnosed at any age. It is extremely heterogeneous, with more than 85 causal genes, and can be inherited in different ways. When a single genetic mutation results in hearing loss, the inheritance is referred to as autosomal dominant. 

An estimated 2,700,000 people in the US have autosomal dominant progressive hearing loss. At this time, there is no effective treatment for these persons. 

We have found allele-specific RNA interference to decrease the rate of autosomal dominant hearing loss. We have identified miRNA plasmids that selectively down-regulate expression of deafness-causing genes. This therapy is specific for autosomal dominant progressive hearing loss. RNA interference selectively decreases expression of only the mutant gene, which in turn is associated with prevention of progression of hearing loss. Proof-of-concept trials were completed in mouse models of hearing loss. The selective nature of the inhibition is an example of precision medicine, as each therapeutic construct is specific for a given mutation thus increases the personalized nature of the therapy.

The World Health Organization estimates that worldwide 500,000,000 people have at least moderate hearing loss. Last year, these persons spent $5.4 billion on treatment. Broadly speaking, their causes of hearing loss fall into two categories: environmental influences and/or genetic factors. Genetic factors are estimated to account for about 50% of hearing loss and are subdivided into syndromic or non-syndromic types of deafness. Syndromic deafness occurs in association with abnormalities in other parts of the body, while non-syndromic deafness is not associated with other signs and symptoms. Non-syndromic deafness accounts for about 80% of genetic hearing loss and can be diagnosed at any age. It is extremely heterogeneous, with more than 85 causal genes, and can be inherited in different ways. When a single genetic mutation results in hearing loss, the inheritance is referred to as autosomal dominant. An estimated 2,700,000 people in the US have autosomal dominant progressive hearing loss. At this time, there is no effective treatment for these persons. We have found allele-specific RNA interference to decrease the rate of autosomal dominant hearing loss. We have identified miRNA plasmids that selectively down-regulate expression of deafness-causing genes. This therapy is specific for autosomal dominant progressive hearing loss. RNA interference selectively decreases expression of only the mutant gene, which in turn is associated with prevention of progression of hearing loss. Proof-of-concept trials were completed in mouse models of hearing loss. The selective nature of the inhibition is an example of precision medicine, as each therapeutic construct is specific for a given mutation thus increases the personalized nature of the therapy.

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  • Cindy C Courterielle —

    Sounds interesting.