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Abstract

Research in Medical & Engineering Sciences

Nano-Gene-Delivery: Overcoming One of the Major Challenges in Gene Therapy

  • Open or Close Sonia Trigueros*

    Department of Zoology, University of Oxford, Englandc

    *Corresponding author: Sonia Trigueros, Oxford Martin Programme of Nanotechnology. Department of Zoology University of Oxford, Oxford, OX1 3PS, England, Email: sonia.trigueros@zoo.ox.ac.uk

Submission: June 12, 2018; Published: July 25, 2018

DOI: 10.31031/RMES.2018.06.000633

ISSN: 2576-8816
Volume6 Issue2

Abstract

Adeno-associated virus vectors (AAVs) have been used in several gene-therapy protocols since they are non-integrative and less immunogenic compared to retroviruses or lentiviruses, but several concerns such as small size capacity, high production costs, probability of immunogenicity, and their invasive route of administration has fostered the design of non-viral vectors as an alternative. Non-viral vectors are less immunogenic, do not induce major inflammatory responses, are safe and easy to produce in large scale. Nonetheless, their main limitation still is the lower cell transfection efficiency. Non-viral particles can be divided into three groups, lipoplex particles containing lipid molecules, polyplex particles based on polycations (sugars, proteins or other polymers), and inorganic nanostructures. Gene-delivery by synthetic vectors involves the endocytosis of DNA-complexes, endosomes escape, and diffusion of non-complexed DNA through the cytosol to reach the nucleus.

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