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Joanna Rejman, Patrick Baumhof; CVCM: A polymeric delivery system for the ocular delivery of mRNA. Invest. Ophthalmol. Vis. Sci. 2018;59(9):5668.
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© ARVO (1962-2015); The Authors (2016-present)
Within the recent year’s nucleic acid-based therapeutics have gained a lot of interest for the treatment of ophthalmic pathologies.
Despite some success of small RNAi based approaches the delivery to the posterior eye segment still remains a major obstacle, especially for larger mRNA. For direct intravitreal injection of nanoparticles the vitreous can interfere with the diffusion of nanoparticles and can prevent reaching the relevant cells.
Here we report a novel polymeric carrier system (CVCMs) that enables the delivery of mRNA by intravitreal and sub-retinal injection to the rat eye. After intravitreal injection of CVCM-encapsulated mRNA, encoding reporter protein, protein expression was detected in eye tissue lysates. After sub-retinal injection of the CVCM encapsulated mRNA the reporter protein expression was localized in the photoreceptor inner segment which is an attractive target tissue for therapeutic intervention.
Overall the CVCM based polymeric delivery system allows for ocular delivery of mRNA and will be utilized to develop therapeutic application in the near future.
This is an abstract that was submitted for the 2018 ARVO Annual Meeting, held in Honolulu, Hawaii, April 29 - May 3, 2018.
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