Pharmaceutical and Biotechnological Applications of Transgenic Technology



  • D Samba Reddy
  • Tina Reddy


A transgenic animal is a genetically modified species in which researchers have modified an existing gene or genes by genetic engineering techniques. Genetic modification involves the mutation, insertion, or deletion of genes. Mouse is the most widely used mammalian species for creating transgenic lines. There are two types of transgenic animals: (i) gene deleted (“knock-out”) and (ii) gene overexpressed (“knock-in”). The loss or gain of gene activity often causes changes in a mouse's phenotype, which includes appearance, behavior and other observable characteristics. Knockout mice are key animal models for studying the role of genes which have been sequenced but whose functions have not been determined.  They include constitutive knockouts (gene deleted since birth) and conditional knockout (gene turned off later after birth).  The first knockout mouse was created in 1989 by Mario Capecchi, Martin Evans, and Oliver Smithies, for which they were awarded the 2007 Nobel Prize in Physiology or Medicine.  Transgenic mouse models have revolutionized the biomedical research and provided a power tool for understanding health and disease. Transgenic animals have been created for bulk production of biotechnology and pharmaceutical products.  In 2009, the FDA approved the first human biological drug ATryn, an anticoagulant extracted from the transgenic goat's milk. The recently discovered CRISPER gene editing technology is providing new frontiers in correcting abnormal genes and hopefully provide cures for genetic diseases in the future.  



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DNA recombination, electroporation, genotyping, chimera, transgenic




How to Cite

Reddy DS, Reddy T. Pharmaceutical and Biotechnological Applications of Transgenic Technology. Scopus Indexed [Internet]. 2018 Jul. 31 [cited 2024 Jun. 20];11(4):4145-53. Available from:



Review Articles


Bai JPF, Alekseyenko AV, Statnikov A, Wang IM and Wong PH (2013). Strategic Applications of Gene Expression: From Drug Discovery/Development to Bedside. AAPS J 15(5): 427-437.
Baron U, Gossen M and Bujard H (1997). Tetracycline-controlled transcription in eukaryotes: novel transactivators with graded transactivation potential. Nucleic Acids Res 25(14): 2723-2729.
Blaese MR, Culver KW, Miller DA, Carter CS, Fleisher T, Clerici M, Shearer G, Chang L, Chiang Y, Tolstoshev P, Greenblatt JJ, Rosenberg SA, Klein H, Berger M, Mullen CA, Ramsey JW, Muul L, Morgan RA and Anderson WF (1995). T lymphocyte-directed gene therapy for ADA- SCID: initial trial results after 4 years. Science 270: 475-480.
Bouabe H and Okkenhaug K (2015). Gene Targeting in Mice: a Review. Methods Mol Biol 1064: 315-336.
Branda CS and Dymecki SM (2004). Talking about a revolution: The impact of site-specific recombinases on genetic analysis in mice. Science Direct 6: 7-28.
Copeland NG, Jenkins NA and Court DL (2001). Recombineering: a powerful new tool for mouse functional genomics. Nature Rev Genetics 2: 769-779.
Deng CX (2012). The Use of Cre–loxP Technology and Inducible Systems to Generate Mouse Models of Cancer. In: J.E. Green and T. Ried (eds.), Genetically Engineered Mice for Cancer Research: Design, Analysis, Pathways, Validation and Pre-Clinical Testing, Springer Science+Business Media, LLC, pp-7.
Dohoo IR, DesCoteaux L, Leslie K, Fredeen A, Shewfelt W, Preston A and Dowling P (2003). A meta-analysis review of the effects of recombinant bovine somatotropin. Can J Vet Res. 67(4): 252-264.
Friedel RH and Soriano P (2017). Gene Trap Mutagenesis in the Mouse. Methods Enzymol. 477: 243-269.
Gierut JJ, Jacks TE and Haigis KM (2014).Strategies to achieve conditional gene mutation in mice. Cold Spring Harb Protoc 2014(4): 339-349.
Giudice A and Trounson A (2008). Genetic Modification of Human Embryonic Stem Cells for Derivation of Target cells. Science Direct 2: 422-433.
Gordon JW (1997). Transgenic Technology and Laboratory Animal Science. ILAR 38(1): 32-41.
Gossler A, Doetschman T, Korn R, Serfling E and Kemler R (1986). Transgenesis by means of blastocyst-derived embryonic stem cell line. Proc. Natl. Acad. Sci. 83(23): 9065-9069.
Hall B, Limaya A and Kulkarni AB (2010). Overview: Generation of Gene Knockout Mice. Curr Protoc Cell Biol 44(1): 1-17.
Houdebine L (2007). Transgenic animal models in biomedical research. Methods in Molecular Biology 360: 163-202.
Hryhorowicz M, Zeyland J, Slomski R and Lipinski D (2017). Genetically Modified Pigs as Organ Donors for Xenotransplantation. Mol Biotechnol 59(9): 435-444.
Ishii A, Kurosawa A, Saito S and Adachi N (2014). Analysis of the Role of Homology Arms in Gene-Targeting Vectors in Human Cells. PLoS ONE 9(9): e108236.
Jaenisch R and Mintz B (1974). Simian Virus 40 DNA Sequences in DNA of Healthy Adult Mice Derived from Preimplantation Blastocysts Injected with Viral DNA. Proc Natl Acad Sci USA 71(4): 1250-1254.
Langford CB and Korneygay JN (2013). Comparative Genomics of X-linked Muscular Dystropies: The Golden Retriever Model. Curr Genomics 14(5): 330-342.
Liu C, Xie W, Gui C and Du Y (2013). Pronuclear Microinjection and Oviduct Transfer Procedures for Transgenic Mouse Production. Methods Mol Biol 1027: 217-232.
Liu P, Jenkins NA and Copeland NG (2003). A highly efficient recombineering-based method for generating conditional knockout methods. Genome Res 13(3): 476-484.
Lo MMS, Conrad MK, Mamalaki C and Kadan MJ (1988). Retroviral-mediated gene transfer. Molecular Neurobiology 2(3): 155-182.
Lobe CG and Nagy A (1998). Conditional genome alteration in mice. Bio Essays 20(3): 200-208.
Lydon JP, DeMayo FJ, Funk CR, Mani SK, Hughes AR, Montgomery CA Jr, Shyamala G, Conneely OM, and O’Malley BW (1995) Mice lacking progesterone receptorexhibit pleiotropic reproductive abnormalities. Genes Dev 9: 2266-2278.
Karreman C (1998). New positive/negative selectable markers for mammalian cells on the basis of blasticidin deaminase-thymidine kinase fusions. Nucleic Acids Res 26(10): 2508-2510.
Maksimenko OG, Deykin AV, Khodarovich YM and Georgiev PG (2013). Use of Transgenic Animals in Biotechnology: Prospects and Problems. Acta Naturae 5(1): 33-46.
Nayerossadat N, Maedah T and Ali PA (2012). Viral and nonviral delivery systems for gene delivery. Adv Biomed Res 1: 27.
Olena T, Michalczechen-Lacerda VA, Rech EL, and Kaplan DL (2013). Recombinant DNA production of spider silk proteins. Microb Biotechnol 6(6): 651-663.
Orban PC, Chui D and Marth JD (1992). Tissue- and site-specific DNA recombination in transgenic mice. Proc. Natl. Acad. Sci. USA 89(15): 6861-6865.
Ormandy EH, Dale J, and Griffin G (2011). Genetic engineering in animals: Ethical issues including welfare concerns. Can Vet J 52(5): 544-550.
Peel CJ, Fronk TJ, Bauman DE and Gorewit RC (1981). Effect of Exogenous Growth Hormone in Early and Late Lactation on Lactational Performance in Dairy Cows. J. Nutr 111: 1662.
Qiao J, Black ME and Caruso M (2000). Enhanced ganciclovir killing and bystander effect of human tumor cells transduced with a retroviral vector carrying a herpes simplex virus thymidine kinase gene mutant. Hum Gene Ther 11(11): 1569-1576.
Rainer NG, Banks GB, Hall JK, Muir LA, Ramos JN, Wicki J, Odom GL, Konieczny P, Seto J, Chamberlain JR and Chamberlain JS (2016). Animal Models of Muscular Dystrophy. Prog Mol Biol Transl Sci 105: 83-111.
Reddy DS, Castaneda DC, O'Malley BW and Rogawski MA (2004). Anticonvulsant activity of progesterone and neurosteroids in progesterone receptor knockout mice. J Pharmacol Exp Ther 310: 230-239.
Tanaka N, Takeuchi T, Neri QV, Sills ES and Palermo GD (2006). Laser-assisted blastocyst dissection and subsequent cultivation of embryonic stem cells in a serum/cell free culture system: applications and preliminary results in a murine model. J Transl Med 4: 20.
Wheeler MB (2013). Transgenic Animals in Agriculture. Nature Education Knowledge 4(11): 1.
Yates F and Daley GQ (2006). Progress and prospects: gene transfer into embryonic stem cells. Gene Therapy 13(20): 1431-1439.
Zhang J, Zhao J, Jiang W, Shan X, Yang X and Gao J (2012). Conditional gene manipulation: Creating a new biological era. J Zhejiang Univ Sci B 13(7): 511-524.

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