CELL FOR GENE THERAPY OF LCAT DEFICIENCY, AND REPLICATION-DEFICIENT RETROVIRUS VECTOR AND PLASMID FOR USE IN THE PRODUCTION OF THE CELL

摘要

Disclosed is a replication-deficient retrovirus vector carrying an LCAT-encoding nucleotide, which enables the LCAT-encoding nucleotide (an exogenous gene) to be expressed in a target cell with efficiency, and which can be used for the production of a cell for gene therapy to be administered to a patient. Also disclosed is a plasmid which can be used for the production of the retrovirus vector. Specifically disclosed is a plasmid for use in the production of a replication-deficient retrovirus vector, which has the 5'-LTR sequence and the 3'-LTR sequence derived from a retrovirus, an LCAT-encoding nucleotide sequence which is inserted betweenthe 5'-LTR sequence and the 3'-LTR sequence as an exogenous gene, and a Kozak's consensus sequence and a packaging signal sequence for the retrovirus both linked to the 5'-end of the LCAT-encoding nucleotide sequence.