Optimized AGA genes and expression cassettes and their use
This invention relates to polynucleotides comprising optimized aspartylglucosaminidase (AGA) open reading frames and vectors and cells comprising the same. The invention further relates to methods of using the same for delivery of the open reading frame to a cell or a subject and methods for treatin...
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Main Author | |
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Format | Patent |
Language | English |
Published |
08.11.2022
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Subjects | |
Online Access | Get full text |
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Summary: | This invention relates to polynucleotides comprising optimized aspartylglucosaminidase (AGA) open reading frames and vectors and cells comprising the same. The invention further relates to methods of using the same for delivery of the open reading frame to a cell or a subject and methods for treating aspartylglucosaminuria (AGU) in a subject. |
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Bibliography: | Application Number: US201816761290 |