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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

Background

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. The increased market could be because of better diagnostics or better access to diagnosis treatments. Moreover, this increase could be due to increased investment, a rise in R and D activities in the field of genetic and inherited disorders and an upsurge in the population. Usher syndrome is one of the many rare genetic disorders and is primarily characterized by deafness accompanied by blindness. As identified by researchers, three clinical types of Usher syndrome, designated as type I, type II, and type

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