The local production of therapeutic proteins has generated considerable interest in the research community as a strategy to overcome the challenges associated with conventional protein-based therapies, such as high costs, the inconvenience of repeated administrations, and unwanted side effects. This review explores the potential of targeted polymeric nanoparticles that carry nucleic acids to induce controlled, localized production of therapeutic antibodies. Polymeric nanoparticles are analyzed on the basis of four key components: i) the polymer matrix, including cationic, non- cationic, lipid-polymer hybrid, and cyclodextrin-based systems; ii) the genetic payload designed for antibody production; iii) linker chemistry, ranging from standard covalent bonds to stimuli- responsive or non-covalent bonds; and iv) potential targeting tools such as antibodies/fragments, peptides, or aptamers, for identifying target cells. By combining gene therapy with nanomedicine, these particles reduce immune responses and show great promise for treating cancer, infections, and autoimmune diseases. To this end, their structure is discussed in relation to the therapeutic antibody’s mechanism of action, and to their in vivo validation as a next-generation platform for antibody-based immunotherapies.

Polymeric Nanoparticles for a Localized In Vivo Production of Therapeutic Antibodies / Frea, M., Durigutto, P., Dal Bo, M., Macor, P.. - In: MOLECULAR THERAPY NUCLEIC ACIDS. - ISSN 2162-2531. - (2026), pp. 1-60. [10.1016/j.omtn.2026.103149]

Polymeric Nanoparticles for a Localized In Vivo Production of Therapeutic Antibodies

Maddalena, Frea
;
Paolo, Durigutto;Paolo, Macor
2026-01-01

Abstract

The local production of therapeutic proteins has generated considerable interest in the research community as a strategy to overcome the challenges associated with conventional protein-based therapies, such as high costs, the inconvenience of repeated administrations, and unwanted side effects. This review explores the potential of targeted polymeric nanoparticles that carry nucleic acids to induce controlled, localized production of therapeutic antibodies. Polymeric nanoparticles are analyzed on the basis of four key components: i) the polymer matrix, including cationic, non- cationic, lipid-polymer hybrid, and cyclodextrin-based systems; ii) the genetic payload designed for antibody production; iii) linker chemistry, ranging from standard covalent bonds to stimuli- responsive or non-covalent bonds; and iv) potential targeting tools such as antibodies/fragments, peptides, or aptamers, for identifying target cells. By combining gene therapy with nanomedicine, these particles reduce immune responses and show great promise for treating cancer, infections, and autoimmune diseases. To this end, their structure is discussed in relation to the therapeutic antibody’s mechanism of action, and to their in vivo validation as a next-generation platform for antibody-based immunotherapies.
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Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/11368/3150718
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