Patent classifications
C
C12
C12Y
108/00
C12Y108/03
C12Y108/03
OPTIMIZED SUMF1 GENES AND EXPRESSION CASSETTES AND THEIR USE
This invention relates to polynucleotides comprising optimized SUMF1 open reading frame (ORF) sequences, vectors comprising the same, and methods of using the same for deliver) of the ORF to a cell or a subject and to treat disorders associated with aberrant expression of a SUMF1 gene or aberrant activity of a SUMF1 gene product in the subject, such as SUMF1 disease.
Optimized SUMF1 genes and expression cassettes and their use
12516301
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2026-01-06
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This invention relates to polynucleotides comprising optimized SUMF1 open reading frame (ORF) sequences, vectors comprising the same, and methods of using the same for deliver) of the ORF to a cell or a subject and to treat disorders associated with aberrant expression of a SUMF1 gene or aberrant activity of a SUMF1 gene product in the subject, such as SUMF1 disease.