Vector compositions and methods of using same for treatment of lysosomal storage disorders
AssigneeM6P Terapeutics (Switzerland) LLC
InventorsCuong Do, Lin Liu
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Abstract
Provided herein are compositions and methods of using a bicistronic vector for treating or preventing a lysosomal storage disorder (LSD) in a subject. The disclosed compositions comprise a bicistronic vector comprising a promoter, an Internal Ribosome Entry Site (IRES), a polynucleotide encoding a lysosomal enzyme and a polynucleotide encoding a modified GlcNAc-1 phosphotransferase (GlcNAc-1 PTase). The present methods comprise administering to the subject a pharmaceutical composition comprising the bicistronic vector as disclosed herein.
Claims15
A composition comprising a vector comprising a sequence encoding a promoter, a first polynucleotide encoding a lysosomal enzyme and a second polynucleotide encoding a modified GlcNAc-1 phosphotransferase (GlcNAc-1 PTase), wherein the promoter is capable of driving expression in a mammalian cell and wherein the promoter is operably linked to the first polynucleotide and to the second polynucleotide, wherein the vector further comprises a sequence encoding an Internal Ribosome Entry Site (IRES), wherein the polynucleotide encoding a modified GlcNAc-1 phosphotransferase comprises a nucleic acid sequence of SEQ ID NO:
- 4.
Dependencies are inferred from the claim text.