Antibody Gene Therapy: Getting Closer to Clinical Application?
Monoclonal antibodies are successfully used in the clinic. However, repeated high-dose bolus injections imply
high costs. As an alternative to recombinant protein administration, gene therapy may provide a novel mean for systemic
delivery of monoclonal antibodies. This strategy has been used in preclinical studies of a wide variety of pathological
conditions, including cancer, infectious diseases, drug addiction, retinal neovascularisation and Alzheimer’s disease. The
two main gene therapy approaches are based on direct gene delivery (using viral or non-viral vectors) or on inoculation of
ex vivo genetically modified cells (autologous or allogenic). Viral vectors are highly efficient as gene delivery vehicles
and have been tested in numerous clinical trials, but still raise concerns about safety and limitation of the therapeutic effect
due to immune responses against viral antigens. On the other hand, use of standard non-viral vectors has been limited
by their low transduction efficiency. Practical application of ex vivo genetically modified cells would imply the availability
of stocks of “ready-to-use” gene-modified allogenic cells that should be protected from the host immune system. Actually,
this field did not meet the expectation raised initially, mainly because of difficulties with obtaining sustained therapeutic
plasma levels in animal models. However, this situation is changing rapidly and the therapeutic potential of these
approaches is getting closer to clinical applications. This review focuses on the current achievements in preclinical studies,
as well as the challenges and future prospects of antibody gene therapy.
Keywords: Antibody, cell factory, gene therapy, immunotherapy, viral vector.
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