Minicircle Delivery to the Neural Retina as a Gene Therapy Approach

Non-viral gene therapy has the potential to overcome several shortcomings in viral vector-based therapeutics. Methods of in vivo plasmid delivery have developed over recent years to increase the efficiency of non-viral gene transfer, yet further improvements still need to be made to improve their tr...

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Bibliographic Details
Published in:International journal of molecular sciences 2022-10, Vol.23 (19), p.11673
Main Authors: Staurenghi, Federica, McClements, Michelle E, Salman, Ahmed, MacLaren, Robert E
Format: Article
Language:English
Subjects:
Deoxyribonucleic acid
Disease
DNA
DNA - genetics
DNA methylation
DNA, Bacterial
Drug delivery
Expression vectors
Gene expression
Gene therapy
Gene Transfer Techniques
Genetic Therapy - methods
Genetic Vectors - genetics
Humans
Immune response
In vivo methods and tests
Innate immunity
minicircle
Nanoparticles
non-viral
Plasmids
Retina
Retinal Diseases
Review
Rodents
Vectors (Biology)
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Summary:Non-viral gene therapy has the potential to overcome several shortcomings in viral vector-based therapeutics. Methods of in vivo plasmid delivery have developed over recent years to increase the efficiency of non-viral gene transfer, yet further improvements still need to be made to improve their translational capacity. Gene therapy advances for inherited retinal disease have been particularly prominent over the recent decade but overcoming physical and physiological barriers present in the eye remains a key obstacle in the field of non-viral ocular drug delivery. Minicircles are circular double-stranded DNA vectors that contain expression cassettes devoid of bacterial DNA, thereby limiting the risks of innate immune responses induced by such elements. To date, they have not been extensively used in pre-clinical studies yet remain a viable vector option for the treatment of inherited retinal disease. Here, we explore the potential of minicircle DNA delivery to the neural retina as a gene therapy approach. We consider the advantages of minicircles as gene therapy vectors as well as review the challenges involved in optimising their delivery to the neural retina.
ISSN:1422-0067
1661-6596
1422-0067
DOI:10.3390/ijms231911673