RECOMBINANT ADENOVIRUS VECTOR FOR TREATING WILSON'S DISEASE

摘要

PROBLEM TO BE SOLVED: To obtain the subject new vector composed of a recombinant adenovirus vector having a gene coding for a copper-transferring protein and a promotor controlling its expression, capable of expressing a copper-transferring protein and useful for the treatment of Wilson's disease, etc. SOLUTION: This new recombinant adenovirus vector, which has a gene coding for a copper-transferring protein and a promotor controlling the expression of the gene, expresses the copper-transferring protein and is useful for gene therapy of Wilson's disease, etc. The recombinant adenovirus vector is obtained by combining a gene coding for a human-derived copper-transferring protein prepared by cloning it using a human liver mRNA as a mold through RT-PCR and a promotor such as CAG-promotor controlling the expression of the gene into a genome deficient of the whole or a part of E1A gene region of adenovirus genome.