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Pharmaceutics
Special Issues
Novel Oligonucleotide Therapeutics and Their Delivery
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Novel Oligonucleotide Therapeutics and Their Delivery

A special issue of Pharmaceutics (ISSN 1999-4923). This special issue belongs to the section "Physical Pharmacy and Formulation".

Deadline for manuscript submissions: closed (30 November 2022) | Viewed by 3283

Special Issue Editors

Dr. Ted Lakowski

E-Mail Website
Guest Editor
College of Pharmacy, University of Manitoba, Apotex Centre, 750 McDermot Ave. W., Winnipeg, MB R3E 0T5, Canada
Interests: drug design; development and delivery; epigenetics; DNA methylation; LC-MS/MS
Dr. Alejandro Martínez-Åguila

E-Mail Website
Guest Editor
R&D Department Avizor Eye Care Solutions, Ocupharm Research Group, Complutense University, 28037 Madrid, Spain
Interests: melatonin; glaucoma; eye care solutions; dry eye; contact lenses
Special Issues, Collections and Topics in MDPI journals

Special Issue Information

Dear Colleagues,

New oligonucleotide therapeutics are becoming increasingly important. Recent approval of parenteral nanoparticle oligonucleotide formulations such as the Pfizer and Moderna COVID RNA vaccines, siRNA therapies such as, givosiran, patisiran, and aptamer therapies like pegaptanib highlight the importance and the increasing success of oligonucleotide delivery. While the promise of antisense oligonucleotide therapies have been anticipated since the late 90s, new oligonucleotide therapies are being produced, including those that induce exon skipping, novel aptamers, and the potential for delivery of gRNA for CRISPR gene editing. All of these promising oligonucleotide technologies converge on an important requirement: effective drug delivery methods that can get the oligonucleotide drugs into cells and target the drug to the right tissues. Nanoparticle delivery technology has advanced to meet some of these challenges and newer technologies are being developed that have extended half-lives, and target nanoparticles to particular tissues using “smart” materials, and chemical conjugation. This Special Issue covers the latest information on novel oligonucleotide therapeutics and their delivery. We are seeking original contributions and review articles in these areas.

Dr. Ted Lakowski
Dr. Alejandro Martínez-Åguila
Guest Editors

Manuscript Submission Information

Manuscripts should be submitted online at www.mdpi.com by registering and logging in to this website. Once you are registered, click here to go to the submission form. Manuscripts can be submitted until the deadline. All submissions that pass pre-check are peer-reviewed. Accepted papers will be published continuously in the journal (as soon as accepted) and will be listed together on the special issue website. Research articles, review articles as well as short communications are invited. For planned papers, a title and short abstract (about 100 words) can be sent to the Editorial Office for announcement on this website.

Submitted manuscripts should not have been published previously, nor be under consideration for publication elsewhere (except conference proceedings papers). All manuscripts are thoroughly refereed through a single-blind peer-review process. A guide for authors and other relevant information for submission of manuscripts is available on the Instructions for Authors page. Pharmaceutics is an international peer-reviewed open access monthly journal published by MDPI.

Please visit the Instructions for Authors page before submitting a manuscript. The Article Processing Charge (APC) for publication in this open access journal is 2900 CHF (Swiss Francs). Submitted papers should be well formatted and use good English. Authors may use MDPI's English editing service prior to publication or during author revisions.

Keywords

  • nanoparticle drug delivery
  • siRNA
  • aptamer
  • anti-sense oligonucleotides
  • bioconjugation
  • miRNA
  • Smart materials
  • CRISPR
  • therapeutic gene editing

Published Papers (1 paper)

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Research

18 pages, 5473 KiB  
Article
Cell-Penetrating Peptide and siRNA-Mediated Therapeutic Effects on Endometriosis and Cancer In Vitro Models
by Kristina Kiisholts, Kaido Kurrikoff, Piret Arukuusk, Ly Porosk, Maire Peters, Andres Salumets and Ülo Langel
Pharmaceutics 2021, 13(10), 1618; https://0-doi-org.brum.beds.ac.uk/10.3390/pharmaceutics13101618 - 05 Oct 2021
Cited by 15 | Viewed by 2664
Abstract
Gene therapy is a powerful tool for the development of new treatment strategies for various conditions, by aiming to transport biologically active nucleic acids into diseased cells. To achieve that goal, we used highly potential delivery vectors, cell-penetrating peptides (CPPs), as oligonucleotide carriers [...] Read more.
Gene therapy is a powerful tool for the development of new treatment strategies for various conditions, by aiming to transport biologically active nucleic acids into diseased cells. To achieve that goal, we used highly potential delivery vectors, cell-penetrating peptides (CPPs), as oligonucleotide carriers for the development of a therapeutic approach for endometriosis and cancer. Despite marked differences, both of these conditions still exhibit similarities, like excessive, uncoordinated, and autonomous cellular proliferation and invasion, accompanied by overlapping gene expression patterns. Thus, in the current study, we investigated the therapeutic effects of CPP and siRNA nanoparticles using in vitro models of benign endometriosis and malignant glioblastoma. We demonstrated that CPPs PepFect6 and NickFect70 are highly effective in transfecting cell lines, primary cell cultures, and three-dimensional spheroids. CPP nanoparticles are capable of inducing siRNA-specific knockdown of therapeutic genes, ribonucleotide reductase subunit M2 (RRM2), and vascular endothelial growth factor (VEGF), which results in the reduction of in vitro cellular proliferation, invasion, and migration. In addition, we proved that it is possible to achieve synergistic suppression of endometriosis cellular proliferation and invasion by combining gene therapy and hormonal treatment approaches by co-administering CPP/siRNA nanoparticles together with the endometriosis-drug danazol. We suggest a novel target, RRM2, for endometriosis therapy and as a proof-of-concept, we propose a CPP-mediated gene therapy approach for endometriosis and cancer. Full article
(This article belongs to the Special Issue Novel Oligonucleotide Therapeutics and Their Delivery)
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