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Nanomedicines to Deliver mRNA: State of the Art and Future Perspectives.

ABSTRACT: The use of messenger RNA (mRNA) in gene therapy is increasing in recent years, due to its unique features compared to plasmid DNA: Transient expression, no need to enter into the nucleus and no risk of insertional mutagenesis. Nevertheless, the clinical application of mRNA as a therapeutic tool is limited by its instability and ability to activate immune responses; hence, mRNA chemical modifications together with the design of suitable vehicles result essential. This manuscript includes a revision of the strategies employed to enhance in vitro transcribed (IVT) mRNA functionality and efficacy, including the optimization of its stability and translational efficiency, as well as the regulation of its immunostimulatory properties. An overview of the nanosystems designed to protect the mRNA and to overcome the intra and extracellular barriers for successful delivery is also included. Finally, the present and future applications of mRNA nanomedicines for immunization against infectious diseases and cancer, protein replacement, gene editing, and regenerative medicine are highlighted.

SUBMITTER: Gomez-Aguado I

PROVIDER: S-EPMC7075285 | biostudies-literature | 2020 Feb

REPOSITORIES: biostudies-literature

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Nanomedicines to Deliver mRNA: State of the Art and Future Perspectives.

Gómez-Aguado Itziar I Rodríguez-Castejón Julen J Vicente-Pascual Mónica M Rodríguez-Gascón Alicia A Solinís María Ángeles MÁ Del Pozo-Rodríguez Ana A

Nanomaterials (Basel, Switzerland) 20200220 2

The use of messenger RNA (mRNA) in gene therapy is increasing in recent years, due to its unique features compared to plasmid DNA: Transient expression, no need to enter into the nucleus and no risk of insertional mutagenesis. Nevertheless, the clinical application of mRNA as a therapeutic tool is limited by its instability and ability to activate immune responses; hence, mRNA chemical modifications together with the design of suitable vehicles result essential. This manuscript includes a revisi ...[more]

PMID: 32093140

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Project description:Current research suggests that the management of neurological diseases, both in adults and children, requires an ever increasing commitment of resources for the national healthcare system (NHS). In Italy, due to the aging of the population, increase in chronicity and morbidity of pathologies, and presence of islands and rural areas, health needs to be supported by innovative technologies. Telemedicine is a method of providing healthcare services at distance, remotely connecting health professionals and patients (or two professionals). In Italy, telemedicine is under development, and the NHS has not yet exploited and independently developed all the possibilities that telemedicine offers. Tele-rehabilitation consists in the use of information and communication technologies for the remote support of rehabilitation services. By allowing “home care,” it represents a valid support during the home rehabilitation process. This review is aimed at evaluating the role of telerehabilitation in Italy, with regard to the motor and cognitive rehabilitation programs applied to neurological pathologies, in both pediatric and adult patients. We screened the studies published between 2010 and 2019 on PubMed, Scopus, Cochrane, and Web of Sciences databases. Using the PICO model, the search combined the terms “telerehabilitation”; “neurological disorders”; “neurodegenerative disease,” “motor telerehabilitation”; “cognitive rehabilitation.” This review showed that telerehabilitation is a promising healthcare tool, as it guarantees continuity of care over time (after discharge) and in space (from hospital to patient's home), especially in patients with stroke. Furthermore, it allows to increase the frequency and intensity of rehabilitation programs, provide individualized rehabilitation treatment in comfortable and familiar environment for patient, monitor and evaluate patients' needs and progress, stimulate patient motivation and achieve better patient satisfaction, verify the results achieved by the patients, and potentially reduce the service costs. Unfortunately, almost all neurorehabilitation studies are characterized by small samples and wide variability of results, and would benefit from standardized procedures, aims and targets. Future telerehabilitation trials should include cost-effectiveness analysis associated with clinical outcomes to better assess the validity of this promising tool.

Dataset Information

Nanomedicines to Deliver mRNA: State of the Art and Future Perspectives.

Publications

Nanomedicines to Deliver mRNA: State of the Art and Future Perspectives.

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