Cell-Delivered Gene TherapyThis Viral Vector Manufacturing Method Could Widen Its Applicability

摘要

Cell-delivered gene therapy is making an impact on a range of diseases (l-17). To date, successful treatments have generally been in conditions involving genetic deficiencies/abnormalities, for which introduction of a normal gene allele has been corrective (l-12,18). Such an approach requires a vector containing the normal allele to overcome the mutant or lacking gene. The vector of choice for cell-delivered gene therapy is often a lentivirus that integrates and expresses introduced therapeutic genesin host target cells and their progeny. Lentiviral vectors have been used for a range of gene therapy applications and in a number of cases have proven to be curative (1-7,11).