Optimized PAH gene and expression cassette and use thereof
AssigneeSuzhou Nggt Biotechnology Co., Ltd.
InventorsShengmei WEN, Yiting LIU, Yufeng GUO, Lixin JIANG
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Abstract
The present disclosure relates to an optimized PAH gene and an expression cassette and use thereof. Specifically, a polynucleotide molecule encoding PAH protein is disclosed. By means of using the polynucleotide molecule, expression cassette, expression vector, virion, and/or pharmaceutical composition provided herein, human PAH can be effectively, persistently, and stably expressed in the liver at a relatively low dose, thus steadily keeping the blood phenylalanine concentration in a subject at a relatively low level for a long period. Therefore, the present disclosure can be used for the treatment of phenylketonuria.
Claims15
A polynucleotide molecule encoding a PAH protein, comprising a nucleotide sequence having 90% or more identity with the nucleotide sequence set forth in SEQ ID NO. 10, SEQ ID NO. 11, SEQ ID NO. 12, SEQ ID NO. 13, SEQ ID NO. 14, SEQ ID NO. 15, SEQ ID NO. 16, SEQ ID NO. 17, SEQ ID NO. 19, SEQ ID NO. 20, SEQ ID NO. 21, SEQ ID NO. 22 or SEQ ID NO. 23;
- preferably, a nucleotide sequence having 91%, 92%, 93%, 94%, 95%, 96%, 97%, 98%, 99% or more identity;
- more preferably, a nucleotide sequence having 98%, 99% or more identity;
- more preferably, the polynucleotide molecule has a nucleotide sequence set forth in SEQ ID NO. 10, SEQ ID NO. 11, SEQ ID NO. 12, SEQ ID NO. 13, SEQ ID NO. 14, SEQ ID NO. 15, SEQ ID NO. 16, SEQ ID NO. 17, SEQ ID NO. 19, SEQ ID NO. 20, SEQ ID NO. 21, SEQ ID NO. 22 or SEQ ID NO. 23.
Dependencies are inferred from the claim text.