METHODS AND COMPOSITIONS FOR INCREASING PROTEIN EXPRESSION AND/OR TREATING A HAPLOINSUFFICIENCY DISORDER

A tRNA that hybridizes to a non-optimal codon can be used to increase expression in a mammalian cell of a gene product encoded by a gene containing the non-optimal codon or to treat a haploinsufficiency disorder in a subject having a haploinsufficient gene containing the non-optimal codon.

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Format Patent
LanguageEnglish
Published 26.09.2019
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Summary:A tRNA that hybridizes to a non-optimal codon can be used to increase expression in a mammalian cell of a gene product encoded by a gene containing the non-optimal codon or to treat a haploinsufficiency disorder in a subject having a haploinsufficient gene containing the non-optimal codon.