Work

Potential and pitfalls of non-viral mRNA delivery for ocular therapies

Year: 2019
Type: dissertation
Abstract: In de field of non-viral gene therapy, in vitro transcribed (IVT) messenger RNA (mRNA) has emerged as a promising tool for the delivery of genetic information.Worldwide effort is committed to the commercial development of mRNA drugs, with ongoing clinical trials focusing on vaccines and cancer immunotherapy.Although the strong immune-stimulatory effect of mRNA is an added benefit for immunotherapeutic vaccination strategies, it is a major disadvantage for non-immunotherapy related applications.The intrinsic immunogenicity of mRNA was shown to directly interfere with the aimed therapeutic outcome of these applications, as it can seriously compromise the expression of the desired protein.Therefore clinical translation of mRNA-therapeutics was seriously hampered in these areas.This chapter presents an overview of the field of mRNA-therapeutics and the immune-related obstacles that limit mRNA advance for non-immunotherapy related applications.In addition, we suggest some promising methods to reduce this 'unwanted' innate immune response and reflect on recent developments in the use of non-viral mRNA delivery for nonimmunogenic purposes.
Language: English
Open Access status: green
OpenAlex ID: W2952585108
Cites: 199
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Related to: 10