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VECTORS COMPRISING A NUCLEIC ACID ENCODING LYSOSOMAL ENZYMES FUSED TO A LYSOSOMAL TARGETING SEQUENCE
VECTORS COMPRISING A NUCLEIC ACID ENCODING LYSOSOMAL ENZYMES FUSED TO A LYSOSOMAL TARGETING SEQUENCE
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机译:包含编码粘合粘合到溶酶体靶向序列的核酸的载体
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摘要
Vectors including viral vectors comprising a genome comprising a heterologous nucleic acid encoding a lysosomal targeting sequence, fused to a lysosomal storage enzyme, enabling the lysosomal enzyme to be targeted to the lysosomes. Particular embodiments relate to a recombinant viral vector, e.g., rAAV vector encoding a lysosomal enzyme, having a lysosomal targeting IGF2(V43) sequence that binds human cation-independent mannose-6-phosphate receptor (CI-MPR) or to the IGF2 receptor, permitting proper subcellular localization of the lysosomal enzyme polypeptide to lysosomes. Also encompassed are therapeutic fusion proteins encoded by the viral vector, non-viral vectors, cells, and methods to treat a glycogen storage disease, e.g., those listed in Table 4A or Table 5A with the viral vector.
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