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Cationic Polymers for the Delivery of Therapeutic Nucleotides

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Abstract

In the last decade, insertion of a therapeutic gene and nucleotide into cells termed as gene therapy has shown tremendous potential to cure life-threatening severe diseases. This therapy involves carrying the therapeutic gene to the nucleus of an affected cell through a vector. Initially viral vectors were employed for this purpose, but low production yield, limited carrying capacity, and long-term safety concerns associated with viral vectors led to the development of nonviral vectors as gene carriers. Currently, used nonviral carriers include polymers and lipids with cationic charge. Cationic polymers provide an excellent alternative for gene delivery due to their water solubility, biodegradability, ease of modification, and excellent compatibility with body systems, but challenges still persist to optimize them as ideal vectors. This chapter provides an overview of present status of cationic polymers and challenges associated with their use for gene delivery.

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Abbreviations

PEI:

Polyethyleneimine

PLL:

Poly-l-lysine

ROS:

Reactive oxygen species

siRNA:

Small interfering RNA

FITC:

Fluorescein isothiocyanate

DNA:

Deoxyribonucleic acid

PEG:

Polyethylene glycol

CD:

Cyclodextrin

SPG:

Schizophyllan

PAMAM:

Poly(amidoamine)

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Authors and Affiliations

  1. Department of Pharmaceutics, National Institute of Pharmaceutical Education and Research (NIPER), Hyderabad, 500037, India

    Anjali Jain & Wahid Khan

  2. Graduate Institute of Biomedical Engineering, National Taiwan University of Science and Technology (TAIWAN TECH), Taipei, 10607, Taiwan

    Hossein Hosseinkhani

  3. School of Pharmacy-Faculty of Medicine, The Hebrew University of Jerusalem and Jerusalem College of Engineering (JCE), Jerusalem, 91120, Israel

    Abraham J. Domb

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  1. Anjali Jain
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  2. Hossein Hosseinkhani
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  3. Abraham J. Domb
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  4. Wahid Khan
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Correspondence to Wahid Khan .

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Editors and Affiliations

  1. Botany Department, M.L.Sukhadia University, Badgaon, India

    Kishan Gopal Ramawat

  2. Institut des Sciences de la Vigne et du Vin, Université de Bordeaux, Villenave d'Ornon, France

    Jean-Michel Mérillon

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Jain, A., Hosseinkhani, H., Domb, A.J., Khan, W. (2014). Cationic Polymers for the Delivery of Therapeutic Nucleotides. In: Ramawat, K., Mérillon, JM. (eds) Polysaccharides. Springer, Cham. https://doi.org/10.1007/978-3-319-03751-6_44-1

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