State-of-Art in mRNA Therapeutics and Gene Delivery

Dear Colleagues,

We are pleased to invite you to participate in this Special Issue, entitled “State-of-the-Art in mRNA Therapeutics and Gene Delivery”. mRNA therapeutics are a new class of drugs and therapeutic strategies based on the insertion of a specific genetic message (mRNA) into the cells and the silencing or gene editing of a specific mRNA. There are several tools available to trigger these mechanisms, such as IVT mRNA, siRNA, shRNA, and miRNA. Given the fast degradation and low intrinsic cell delivery efficiency that some of these molecules exhibit, gene delivery vectors play a very significant role in the therapeutic efficiency of mRNA-based therapies. There are two main groups of delivery vectors: viral vectors, such as the lentiviral and adeno-associated viral particles; and non-viral vectors, such as lipid- and polymer-based nanoparticles. Numerous preclinical studies and some clinical trials based on mRNA therapeutics were launched with the aim of treating  various human diseases, and applied to fields such as cancer immunotherapy, infectious disease vaccines, protein replacement, genome engineering, and the genetic reprogramming of cells.

This Special Issue aims to provide an overview of the current state of mRNA-based therapeutics, namely the available therapeutic molecules and delivery vectors, their modes of action, and the preclinical and clinical testing of new mRNA-based therapeutics. This issue will cover some of the main critical aspects of mRNA-based therapeutics development; the latest advances, challenges and strengths; and various experimental and therapeutic applications.

In this Special Issue, original research articles and reviews are welcome. Research areas may include (but are not limited to) the following:

• Advances in mRNA-based gene therapy;
• Therapeutic oligonucleotides;
• Short-hairpin RNA (shRNA), small interfering RNA (siRNA), microRNA (miRNA), small activating RNA (saRNA), exon-skipping antisense oligonucleotides;
• In vitro transcribed (IVT) mRNA, mRNA-based protein replacement therapy, mRNA-based vaccines, mRNA-mediated antibody expression;
• The preclinical development and clinical testing of tools and techniques for mRNA-based therapies;
• The preclinical development and clinical testing of non-viral and viral vectors for mRNA-based therapies delivery.

We look forward to receiving your contributions.

Dr. Liliana Mendonca
Guest Editor

Manuscript Submission Information

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