Use of Transgenic Animals in Biotechnology: Prospects and Problems

Abstract

During the past two decades, there have been numerous attempts at using animals in order to produce recombinant human proteins and monoclonal antibodies. However, it is only recently that the first two therapeutic agents isolated from the milk of transgenic animals, C1 inhibitor (Ruconest) and antithrombin (ATryn), appeared on the market. This inspires hope that a considerable number of new recombinant proteins created using such technology could become available for practical use in the near future. In this review, the methods applied to produce transgenic animals are described and the advantages and drawbacks related to their use for producing recombinant human proteins and monoclonal antibodies are discussed.

Recombinant human C1-esterase inhibitor relieves symptoms of hereditary angioedema attacks: phase 3, randomized, placebo-controlled trial

“…8e12 In addition to the advantages presented by transgenic animals, including the ability to produce active proteins exhibiting complex post-translational modifications (eg, glycosylation, cleavage, folding), the transgenic rabbit platform ensures a reliable and scalable supply of product of uniform quality that is not dependent on human plasma donors. 7,13 The efficacy and safety of rhC1INH in the treatment of HAE symptoms were evaluated in 2 independent, randomized, placebocontrolled, double-blinded studies and 2 open-label extension studies. In the randomized controlled trials, patients treated with rhC1INH 50 IU/kg (12 patients) or 100 IU/kg (29 patients) had significantly shorter times to beginning of relief from symptoms and to minimal symptoms, assessed using visual analog scales (VASs), compared with placebo-treated patients.…”

Recombinant Human-C1 Inhibitor Is Effective and Safe for Repeat Hereditary Angioedema Attacks

“…In addition, the transgenic rabbit platform ensures a reliable and scalable supply of a product with uniform quality. 9 The recombinant protein, with a sequence identical to that of human C1INH, is expressed in mammary glands of transgenic rabbits and purified from milk. Because rhC1INH retains the specificity of human pdC1INH toward its target proteases, the mode of action is identical.…”

Production of functional human nerve growth factor from the saliva of transgenic mice by using salivary glands as bioreactors

“…Therefore, transgenic (TG) animals have been employed for the production of various therapeutically important human proteins1234. To date, two therapeutic proteins produced from the milk of TG animals have been approved for commercial and clinical use in Europe and the USA2.…”