Abstract
MicroRNAs (miRNAs) are short (~21–23 nucleotides), non-coding endogenous RNA molecules that modulate gene expression at the post-transcriptional level via the endogenous RNA interference machinery of the cell. They have emerged as potential biopharmaceuticals candidates for the treatment of various diseases, including cancer, cardiovascular and metabolic diseases. However, in order to advance miRNAs therapeutics into clinical settings, their delivery remains a major challenge. Different types of vectors have been investigated to allow the delivery of miRNA in the diseased tissue. In particular, non-viral delivery systems have shown important advantages such as versatility, low cost, easy fabrication and low immunogenicity. Here, we present a general overview of the main types of non-viral vectors developed for miRNA delivery, with their advantages, limitations and future perspectives.
Highlights
LVTS, INSERM U1148, Université Sorbonne Paris Nord, Université de Paris, 75018 Paris, France; Centre de Biophysique Moléculaire, CNRS UPR4301, Université d’Orléans, 45071 Orléans, France; Present address: Nanobiotix, 60 Rue de Wattignies, 75012 Paris, France
The development of innovative therapies based on nucleic acids and proteins has emerged as highly specific pharmaceutical agents [1,2]
We provide a general overview of the different classes of non-viral carriers developed for miRNA delivery, their advantages and limitations, their therapeutic applications, as well as future perspectives in the design and development of miRNA
Summary
The development of innovative therapies based on nucleic acids (including plasmid DNA, antisense oligonucleotides, messenger RNAs, siRNAs, miRNAs, etc.) and proteins (monoclonal antibodies, growth factors, hormones, therapeutic enzymes, synthetic oligopeptides, etc.) has emerged as highly specific pharmaceutical agents [1,2]. MiRNA mimics (replacement therapy) is employed when the target miRNA is down-regulated This replacement could be obtained as well with the use of small synthetic double-stranded molecules processed into functional miRNA or miRNA expression vectors to induce expression of a miRNA in cells and the delivery of the miRNA itself. The main obstacle preventing the implementation of miRNA-based therapies in clinical practice is the lack of an efficient delivery system, which is the case of other nucleic acids therapeutics Despite their small size, miRNAs are different from conventional drugs in that they cannot passively diffuse across lipid membranes into target cells and exhibit very limited biological stability [35]. We provide a general overview of the different classes of non-viral carriers developed for miRNA delivery, their advantages and limitations, their therapeutic applications, as well as future perspectives in the design and development of miRNA delivery technologies
Sign-in/Register to view highlights & summary Talk to us
Join us for a 30 min session where you can share your feedback and ask us any queries you have
Schedule a callDisclaimer: All third-party content on this website/platform is and will remain the property of their respective owners and is provided on "as is" basis without any warranties, express or implied. Use of third-party content does not indicate any affiliation, sponsorship with or endorsement by them. Any references to third-party content is to identify the corresponding services and shall be considered fair use under The CopyrightLaw.
