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Antisense Oligonucleotides DFNA9

The AONs may be used in therapy for inherited deafness, ophthalmology, and Stargardt disease

Background

DFNA9 is a relatively common form of adult-onset, autosomal dominantly inherited, progressive hearing loss. DFNA9 is caused by mutations in the COCH gene, and leads to complete deafness by the age of 50-70. DFNA9 patients furthermore suffer from severe balance loss, hampering their daily movements. Dominant COCH mutations result in the formation of cytotoxic protein aggregates, consisting of both mutant and normal COCH proteins, that interfere with hearing and balance function.

Currently, no effective treatment is available to prevent or delay the loss of hearing and balance problems in DFNA9 patients. Antisense oligonucleotides (AONs) are small synthetic single-stranded DNA/RNA molecules that can be used target pre-mRNA for degradation. In a dominantly inherited disorder such as DFNA9, they can be

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