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Review
. 2019:1148:25-54.
doi: 10.1007/978-981-13-7709-9_2.

Production of Therapeutic Enzymes by Lentivirus Transgenesis

María Celeste Rodríguez  1 , Natalia Ceaglio  1 , Sebastián Antuña  2 , María Belén Tardivo  2 , Marina Etcheverrigaray  1 , Claudio Prieto  3
Affiliations
Review

Production of Therapeutic Enzymes by Lentivirus Transgenesis

María Celeste Rodríguez et al. Adv Exp Med Biol. 2019.

Abstract

Since ERT for several LSDs treatment has emerged at the beginning of the 1980s with Orphan Drug approval, patients' expectancy and life quality have been improved. Most LSDs treatment are based on the replaced 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 (rhαGAL), which is used as ERT for Fabry disease treatment.

Keywords: ERT; Fabry disease; LSDs; Lentiviral Vectors (LVs); rhαGAL.

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References

    1. Bandaranayake AD, Almo SC (2014) Recent advances in mammalian protein production. FEBS Lett 588(2):253–260 - PubMed
    1. Baranyi L, Roy A, Embree HD, Dropulic B (2010) Lentiviral vector-mediated genetic modification of cell substrates for the manufacture of proteins and other biologics. PDA J Pharm Sci Technol 64:379–385 - PubMed
    1. Bennett LL, Mohan D (2013) Gaucher disease and its treatment options. Ann Pharmacother 47(9):1182–93
    1. Braulke T, Bonifacino JS (2009) Sorting of lysosomal proteins. BBA – Mol Cell Res 1793:605–614
    1. Braunlin E, Rosenfeld H, Kampmann C et al (2013) Enzyme replacement therapy for mucopolysaccharidosis VI: long-term cardiac effects of galsulfase (Naglazyme®) therapy. J Inherit Metab Dis 36(2):385–394 - PubMed

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