Gene therapy for sulfatase deficiency
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.
Claims49
-85. (canceled)
A gene therapy method of treating a sulfatase deficiency in a subject, the method comprising delivering to a cell of the subject a vector comprising a polynucleotide encoding a Formylglycine Generating Enzyme (FGE) comprising an amino acid sequence having at least 80% identity to the amino acid sequence of amino acids 34-374 of SEQ ID NO:2, wherein the FGE has Cα-formylglycine generating activity.
A composition for gene therapy for a sulfatase deficiency comprising a vector comprising a polynucleotide encoding a Formylglycine Generating Enzyme (FGE) comprising an amino acid sequence having at least 80% identity to the amino acid sequence of amino acids 34-374 of SEQ ID NO:2, wherein the FGE has Cα-formylglycine generating activity.
A gene therapy vector comprising a polynucleotide encoding a Formylglycine Generating Enzyme (FGE) comprising an amino acid sequence having at least 80% identity to the amino acid sequence of amino acids 34-374 of SEQ ID NO:2, wherein the FGE has Cα-formylglycine generating activity.
A cell transduced with a gene therapy vector comprising a polynucleotide encoding a Formylglycine Generating Enzyme (FGE) comprising an amino acid sequence having at least 80% 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.