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Antisense Oligonucleotides (ASO) as a Therapeutic for Usher Syndrome

A therapeutic treatment of Usher Syndrome using ASO targeting RNA transcription of the USH1c gene


Usher syndrome is the leading genetic cause of combined blindness and deafness. It has no known cure. It affects approximately 4 in every 100,000 births. This high growth may be attributed to the rise of inherited disorders. Another driving factor is there is an increase in genetic and inherited diseases. The increased market could be because of better diagnostics or better access to diagnosis treatments. Moreover, this increase could be due to increased investment, rise in R and D activities in the field of genetic and inherited disorders and upsurge in the population.

Usher Syndrome is an inherited disease caused due to loss of a gene suggesting that a correction of gene expression would be a therapeutic treatment option.

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