The Delivery of RNA-Based Therapeutics: New Strategies in Cancer and Gene Therapy

A special issue of Pharmaceutics (ISSN 1999-4923). This special issue belongs to the section "Gene and Cell Therapy".

Deadline for manuscript submissions: 20 May 2024 | Viewed by 13133

Special Issue Editors

1. Department of Biochemistry, Medical Research Institute, Alexandria University, Alexandria 21561, Egypt
2. IBMM, University of Montpellier, CNRS, ENSCM, 34093 Montpellier, France
Interests: nanomedicine; nanomaterials; photodynamic therapy; photothermal therapy; RNA delivery; drug delivery; photochemical internalization; biochemistry; toxicology
IBMM, Univ. Montpellier, CNRS, ENSCM, 34093 Montpellier, France
Interests: cancer research; biotechnology; cell biology
Special Issues, Collections and Topics in MDPI journals
IBMM, University of Montpellier, CNRS, ENSCM, 34093 Montpellier, France
Interests: photodynamic therapy; two-photon excitation; cancer targeting
Special Issues, Collections and Topics in MDPI journals

Special Issue Information

Dear Colleagues,

Ribonucleic acids (RNAs) are important molecular structures that are involved in many functions in cell machinery, including protein synthesis, RNA editing, gene regulation, and RNA interference. Since the fifties of the last century, discoveries are in progress with great promises in RNA therapeutics. Thus far, several RNA-based therapeutics, including antisense oligonucleotides, RNA interference, RNA aptamers, and messenger RNAs, have been approved by the FDA. Nevertheless, their delivery is limited to their encapsulation in lipid-based nanoparticles, in addition of targeting moieties or direct delivery without any modifications. Therefore, the delivery of RNA therapeutics is one of the most controversial and researched topics in RNA therapy. This Special Issue aims to discuss innovative delivery systems and techniques in RNA-based therapeutics to fight cancer and other diseases. In this Special Issue, original research articles and reviews are welcome.

We look forward to receiving your contributions.

Dr. Lamiaa M.A. Ali
Dr. Nadir Bettache
Dr. Magali Gary-Bobo
Guest Editors

Manuscript Submission Information

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Keywords

  • RNA delivery
  • cancer
  • nanoparticles
  • RNA interference
  • gene therapy
  • mRNA
  • photochemical internalization
  • encapsulation
  • delivery

Published Papers (4 papers)

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Research

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23 pages, 5070 KiB  
Article
Novel Tripodal Polyamine Tris-Pyrene: DNA/RNA Binding and Photodynamic Antiproliferative Activity
by Marta Jurković, Marijana Radić Stojković, Ksenija Božinović, Davor Nestić, Dragomira Majhen, Estefanía Delgado-Pinar, Mario Inclán, Enrique García-España and Ivo Piantanida
Pharmaceutics 2023, 15(9), 2197; https://doi.org/10.3390/pharmaceutics15092197 - 25 Aug 2023
Viewed by 819
Abstract
A novel tri-pyrene polyamine (TAL3PYR) bearing net five positive charges at biorelevant conditions revealed strong intramolecular interactions in aqueous medium between pyrenes, characterised by pronounced excimer fluorescence. A novel compound revealed strong binding to ds-DNA and ds-RNA, along with pronounced thermal [...] Read more.
A novel tri-pyrene polyamine (TAL3PYR) bearing net five positive charges at biorelevant conditions revealed strong intramolecular interactions in aqueous medium between pyrenes, characterised by pronounced excimer fluorescence. A novel compound revealed strong binding to ds-DNA and ds-RNA, along with pronounced thermal stabilisation of DNA/RNA and extensive changes in DNA/RNA structure, as evidenced by circular dichroism. New dye caused pronounced ds-DNA or ds-RNA condensation, which was attributed to a combination of electrostatic interactions between 5+ charge of dye and negatively charged polynucleotide backbone, accompanied by aromatic and hydrophobic interactions of pyrenes within polynucleotide grooves. New dye also showed intriguing antiproliferative activity, strongly enhanced upon photo-induced activation of pyrenes, and is thus a promising lead compound for theranostic applications on ds-RNA or ds-DNA targets, applicable as a new strategy in cancer and gene therapy. Full article
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14 pages, 3752 KiB  
Article
The Development of Cell-Penetrating Peptides for Efficient and Selective In Vivo Expression of mRNA in Spleen Tissue
by Ly Porosk, Heleri Heike Härk, Piret Arukuusk, Uku Haljasorg, Pärt Peterson and Kaido Kurrikoff
Pharmaceutics 2023, 15(3), 952; https://doi.org/10.3390/pharmaceutics15030952 - 15 Mar 2023
Cited by 5 | Viewed by 2271
Abstract
mRNA-based therapeutics are presently one of the nucleic acid-based therapeutics with a high potential for extraordinary success as preventive vaccines. Current applications with mRNA therapeutics rely on lipid nanoparticle (LNP) mediated delivery of nucleic acids. In order to achieve the transition from preventive [...] Read more.
mRNA-based therapeutics are presently one of the nucleic acid-based therapeutics with a high potential for extraordinary success as preventive vaccines. Current applications with mRNA therapeutics rely on lipid nanoparticle (LNP) mediated delivery of nucleic acids. In order to achieve the transition from preventive to therapeutic vaccines, there is a challenge of delivering the mRNA into non-hepatic tissues, especially into lymphoid tissues such as the spleen and lymph nodes. In this work, we characterize new cell-penetrating peptides NF424 and NF436 that exhibit preferential delivery of mRNA into the spleen after a single i.v. injection, without the use of any active targeting mechanisms. We show that between the spleen, liver, and the lungs, >95% of mRNA expression arises in the spleen tissue and the majority of expression occurs in the dendritic cells. The cell-penetrating peptides NF424 and NF436 represent promising candidates for cancer immunotherapeutic applications with tumor antigens. Full article
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Review

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30 pages, 5584 KiB  
Review
Unraveling Therapeutic Opportunities and the Diagnostic Potential of microRNAs for Human Lung Cancer
by Osama Sweef, Elsayed Zaabout, Ahmed Bakheet, Mohamed Halawa, Ibrahim Gad, Mohamed Akela, Ehab Tousson, Ashraf Abdelghany and Saori Furuta
Pharmaceutics 2023, 15(8), 2061; https://doi.org/10.3390/pharmaceutics15082061 - 31 Jul 2023
Cited by 5 | Viewed by 1556
Abstract
Lung cancer is a major public health problem and a leading cause of cancer-related deaths worldwide. Despite advances in treatment options, the five-year survival rate for lung cancer patients remains low, emphasizing the urgent need for innovative diagnostic and therapeutic strategies. MicroRNAs (miRNAs) [...] Read more.
Lung cancer is a major public health problem and a leading cause of cancer-related deaths worldwide. Despite advances in treatment options, the five-year survival rate for lung cancer patients remains low, emphasizing the urgent need for innovative diagnostic and therapeutic strategies. MicroRNAs (miRNAs) have emerged as potential biomarkers and therapeutic targets for lung cancer due to their crucial roles in regulating cell proliferation, differentiation, and apoptosis. For example, miR-34a and miR-150, once delivered to lung cancer via liposomes or nanoparticles, can inhibit tumor growth by downregulating critical cancer promoting genes. Conversely, miR-21 and miR-155, frequently overexpressed in lung cancer, are associated with increased cell proliferation, invasion, and chemotherapy resistance. In this review, we summarize the current knowledge of the roles of miRNAs in lung carcinogenesis, especially those induced by exposure to environmental pollutants, namely, arsenic and benzopyrene, which account for up to 1/10 of lung cancer cases. We then discuss the recent advances in miRNA-based cancer therapeutics and diagnostics. Such information will provide new insights into lung cancer pathogenesis and innovative diagnostic and therapeutic modalities based on miRNAs. Full article
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24 pages, 1151 KiB  
Review
Recent Advancement in mRNA Vaccine Development and Applications
by Nojoud Al Fayez, Majed S. Nassar, Abdullah A. Alshehri, Meshal K. Alnefaie, Fahad A. Almughem, Bayan Y. Alshehri, Abdullah O. Alawad and Essam A. Tawfik
Pharmaceutics 2023, 15(7), 1972; https://doi.org/10.3390/pharmaceutics15071972 - 18 Jul 2023
Cited by 6 | Viewed by 7782
Abstract
Messenger RNA (mRNA) vaccine development for preventive and therapeutic applications has evolved rapidly over the last decade. The mRVNA vaccine has proven therapeutic efficacy in various applications, including infectious disease, immunotherapy, genetic disorders, regenerative medicine, and cancer. Many mRNA vaccines have made it [...] Read more.
Messenger RNA (mRNA) vaccine development for preventive and therapeutic applications has evolved rapidly over the last decade. The mRVNA vaccine has proven therapeutic efficacy in various applications, including infectious disease, immunotherapy, genetic disorders, regenerative medicine, and cancer. Many mRNA vaccines have made it to clinical trials, and a couple have obtained FDA approval. This emerging therapeutic approach has several advantages over conventional methods: safety; efficacy; adaptability; bulk production; and cost-effectiveness. However, it is worth mentioning that the delivery to the target site and in vivo degradation and thermal stability are boundaries that can alter their efficacy and outcomes. In this review, we shed light on different types of mRNA vaccines, their mode of action, and the process to optimize their development and overcome their limitations. We also have explored various delivery systems focusing on the nanoparticle-mediated delivery of the mRNA vaccine. Generally, the delivery system plays a vital role in enhancing mRNA vaccine stability, biocompatibility, and homing to the desired cells and tissues. In addition to their function as a delivery vehicle, they serve as a compartment that shields and protects the mRNA molecules against physical, chemical, and biological activities that can alter their efficiency. Finally, we focused on the future considerations that should be attained for safer and more efficient mRNA application underlining the advantages and disadvantages of the current mRNA vaccines. Full article
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