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1c) and preliminary mutational studies on Tyr110 show indeed similar effects to Lys105 (Supplementary Figs 18 and 19)
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It's one of the important lipids that can be found naturally in the outer layer of the skin
This drug aims to halt or reverse vision loss by restoring the function of the usherin protein through exon skipping in Usher syndrome type 2a and non-syndromic retinitis pigmentosa
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