Production of therapeutic enzymes by lentivirus transgenesis

Abstract

Since ERT for several RSDs treatment has emerged at the beginning of the 1980s with Orphan Drug approval, patients´s expectancy and life quality have been improved. Most LSDs treatment are based on the replacement of mutated or deficient protein with the natural or recombinant protein.One of the main ERT drawback is the high drug prices. Therefore, different strategies trying to optimize the global ERT biotherapeutic production have been proposed. LVs, a gene delivery tool, can be proposed as an alternative method to generate stable cell lines in manufacturing of recombinant proteins. Since LVs have been used in human gene therapy, clinical trials, safety testing assays and procedures have been developed. Moreover, one of the main advantages of LVs strategy to obtain manufacturing cell line is the short period required as well as the high protein levels achieved.In this chapter, we will focus on LVs as a recombinant protein production platform and we will present a case study that employs LVs to express in a manufacturing cell line, alpha-Galactosidase A (rhaGAL), whic is used as ERT for Fabry disease treatment.