porphobilinogen deaminase gene therapy

摘要

Use of a parvovirus virion comprising: a) a nucleic acid construct comprising: i) a nucleotide sequence encoding a human porphobilinogen deaminase and having the nucleotide sequence of SEQ ID NO: 1; and, ii) an AAT promoter combined with the mouse albumin gene enhancer element (Ealb); and, iii) AAV ITRs of serotype 2; and, b) AAV capsid proteins of serotype 5; for the production of a medicament for the treatment of a condition caused by a deficiency of porphobilinogen deaminase.