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Gene and Ribonucleic Acid Therapy

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Fundamentals of Pharmaceutical Nanoscience

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Abstract

Gene and small interfering ribonucleic acid (siRNA) therapies are applicable to a wide range of diseases; diseases where a mutated gene is the basis for the disease, e.g. cystic fibrosis, haemophilia and cancer. However, there are some difficulties with gene and siRNA therapy, i.e. low gene and siRNA internalisation into the cells, instability of the nucleic acid molecule in the cell and the lack of specific gene targeting to the nucleus. Although physical methods exist to deliver naked deoxyribonucleic acid (DNA) into cell nuclei, these methods are inefficient and hence nucleic acid vectors are used. These delivery vectors are viruses, cationic polymers and dendrimers or cationic liposomes; with the synthetic vectors all possessing a mandatory amine functional group. Viral vectors are the most efficient gene vectors and there are currently three licenced products available: Rexin G for the treatment of malignant solid tumours, Gendicine for the treatment of head and neck cancer and Glybera for the treatment of lipoprotein lipase deficiency; these three are gene therapies involving viral vectors. Other nucleic acid delivery vectors that are still in development include amine polymers, dendrimers and liposomes prepared from amine phospholipids.

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Authors and Affiliations

  1. UCL School of Pharmacy, University College London, 29‐39, Brunswick Square, London, WC1N 1AX, UK

    Jayanant Iemsam-Arng, Andreas G. Schätzlein & Ijeoma F. Uchegbu

  2. Department of Pharmaceutical Sciences, King’s College London, Strand, London, WC2R 2LS, UK

    Xiao Kong

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  1. Jayanant Iemsam-Arng
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  2. Xiao Kong
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  3. Andreas G. Schätzlein
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  4. Ijeoma F. Uchegbu
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Correspondence to Ijeoma F. Uchegbu .

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  1. UCL School of Pharmacy, University College London, London, United Kingdom

    Ijeoma F. Uchegbu

  2. UCL School of Pharmacy, University College London, London, United Kingdom

    Andreas G. Schätzlein

  3. Bexleyheath, United Kingdom

    Woei Ping Cheng

  4. School of Pharmacy & Biomedical Sciences, University of Portsmouth, Portsmouth, United Kingdom

    Aikaterini Lalatsa

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Iemsam-Arng, J., Kong, X., Schätzlein, A.G., Uchegbu, I.F. (2013). Gene and Ribonucleic Acid Therapy. In: Uchegbu, I., Schätzlein, A., Cheng, W., Lalatsa, A. (eds) Fundamentals of Pharmaceutical Nanoscience. Springer, New York, NY. https://doi.org/10.1007/978-1-4614-9164-4_18

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