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Transgenic Res. 2016 Jun;25(3):329-43. doi: 10.1007/s11248-016-9933-9. Epub 2016 Jan 28.

The transgenic animal platform for biopharmaceutical production.

Author information

1
Department of Pharmacology, Pontifical Catholic University of Rio Grande do Sul (PUC/RS), Porto Alegre, RS, Brazil. luciana.bertolini@pucrs.br.
2
Molecular and Developmental Biology Lab, Health Sciences Center, University of Fortaleza (UNIFOR), Fortaleza, CE, Brazil. luciana.bertolini@pucrs.br.
3
LFB, USA, Framingham, MA, USA.
4
Molecular and Developmental Biology Lab, Health Sciences Center, University of Fortaleza (UNIFOR), Fortaleza, CE, Brazil.
5
Embryology and Reproductive Biotechnology Lab, School of Veterinary Medicine, Federal University of Rio Grande do Sul (UFRGS), Porto Alegre, RS, Brazil.
6
Transgenics Lab, Department of Animal Science, University of California, Davis (UC Davis), Davis, CA, USA.

Abstract

The recombinant production of therapeutic proteins for human diseases is currently the largest source of innovation in the pharmaceutical industry. The market growth has been the driving force on efforts for the development of new therapeutic proteins, in which transgenesis emerges as key component. The use of the transgenic animal platform offers attractive possibilities, residing on the low production costs allied to high productivity and quality of the recombinant proteins. Although many strategies have evolved over the past decades for the generation of transgenic founders, transgenesis in livestock animals generally faces some challenges, mainly due to random transgene integration and control over transgene copy number. But new developments in gene editing with CRISPR/Cas system promises to revolutionize the field for its simplicity and high efficiency. In addition, for the final approval of any given recombinant protein for animal or human use, the production and characterization of bioreactor founders and expression patterns and functionality of the proteins are technical part of the process, which also requires regulatory and administrative decisions, with a large emphasis on biosafety. The approval of two mammary gland-derived recombinant proteins for commercial and clinical use has boosted the interest for more efficient, safer and economic ways to generate transgenic founders to meet the increasing demand for biomedical proteins worldwide.

KEYWORDS:

Biopharmaceutic products; Biopharming; Expression system platforms; Genetic engineering; Genetically modified organisms; Recombinant protein; Transgenesis

PMID:
26820414
DOI:
10.1007/s11248-016-9933-9
[Indexed for MEDLINE]

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