C12Y108/03

OPTIMIZED SUMF1 GENES AND EXPRESSION CASSETTES AND THEIR USE
20220213450 · 2022-07-07 ·

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

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.