Therapeutic strategies to treat CNS pathologies in mucopolysaccharidosis

摘要

A nucleotide sequence encoding a chimeric sulphatase, said chimeric sulfatase consisting of the order of N-terminal-C-terminal sequence: a) a signal peptide or either the human α-antitrypsin amino acid sequence (hAAT) or the amino acid sequence of human iduronate-2-sulphatase (IDS), said sequence belonging to the following group of SEQ ID No. 2 or SEQ ID No. 4; b) an amino acid sequence of human sulfatase devoid of its signal peptide and c) the LDLR binding domain of ApoB.