Cell that expresses a sulfatase and a formylglycine generating enzyme
AssigneeShire Human Genetic Therapies, Inc.
InventorsKurt von Figura, Bernhard Schmidt, Thomas Dierks, Michael W. Heartlein, Maria Pia Cosma, Andrea Ballabio
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Abstract
This invention relates to methods and compositions for the diagnosis and treatment of Multiple Sulfatase Deficiency (MSD) as well as other sulfatase deficiencies. More specifically, the invention relates to isolated molecules that modulate post-translational modifications on sulfatases. Such modifications are essential for proper sulfatase function.
Claims22
An isolated prokaryotic cell transformed with a vector comprising a heterologous polynucleotide encoding a cysteine-type sulfatase and transformed with a vector comprising a polynucleotide encoding a Formylglycine Generating Enzyme (FGE) comprising an amino acid sequence having at least 95% identity to the amino acid sequence of amino acids 34-374 of SEQ ID NO:2, wherein the FGE has Cα-formylglycine generating activity.
An isolated eukaryotic cell transfected with a vector comprising a heterologous polynucleotide encoding a cysteine-type sulfatase and further transfected with a vector comprising a heterologous polynucleotide encoding a Formylglycine Generating Enzyme (FGE) comprising an amino acid sequence having at least 95% identity to the amino acid sequence of amino acids 34-374 of SEQ ID NO:2, wherein the FGE has Cα-formylglycine generating activity.
Dependencies are inferred from the claim text.