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Pharmaceutics
Special Issues
Delivery of Oligonucleotide Therapeutics
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Delivery of Oligonucleotide Therapeutics

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

Deadline for manuscript submissions: closed (20 December 2024) | Viewed by 1733

Special Issue Editors

Dr. Domenica Musumeci

E-Mail Website
Guest Editor
Department of Chemical Sciences, University of Naples Federico II, 80126 Naples, Italy
Interests: oligonucleotide aptamers; oligonucleotide analogs; nucleopeptides; metal-based drugs; DNA-small molecule interaction; DNA-protein interaction
Special Issues, Collections and Topics in MDPI journals
Dr. Ettore Napolitano

E-Mail Website
Guest Editor
Department of Chemical Sciences, University of Naples Federico II, 80126 Naples, Italy
Interests: noncanonical nucleic acids; nucleic acid theranostics; small molecule–DNA interactions
Dr. Anna Di Porzio

E-Mail Website
Guest Editor
Department of Pharmacy, University of Naples Federico II, 80131 Naples, Italy
Interests: noncanonical nucleic acids; cellular and molecular biology; medicinal chemistry
Special Issues, Collections and Topics in MDPI journals

Special Issue Information

Dear Colleagues,

Oligonucleotide therapeutics (OTs) are synthetic DNA or RNA strands with the potential to treat a wide range of diseases. OTs include several molecular entities, such as antisense oligonucleotides (ASOs), small interfering RNAs (siRNAs), anti‐microRNAs (antagomirs), microRNA mimics (agomirs), aptamers and unmethylated CpG‐containing oligonucleotides.

Until now, fifteen FDA-approved oligonucleotide drugs have been released on the market, with many other OTs being investigated.

OTs are promising candidates in the pharmaceutical context. Nevertheless, one of the greatest challenges in oligonucleotide therapy is the delivery of the active substances to the site of action, mainly due to their unfavorable pharmacokinetic profile and nuclease degradation.

To overcome these issues, many strategies have been reported in the literature, ranging from chemical modifications of the DNA/RNA backbone to bioconjugation or encapsulation in nanocarriers.

The continuous progress in this field is gradually unlocking the true potential of oligonucleotide-based therapeutics, opening new perspectives and expanding the range of possible applications.

Authors are invited to submit original papers, communications and reviews regarding the delivery of oligonucleotide therapeutics, to be published in this Special Issue of Pharmaceutics.

Dr. Domenica Musumeci
Dr. Ettore Napolitano
Dr. Anna Di Porzio
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

  • DNA
  • RNA
  • oligonucleotides
  • nucleic acid-based therapeutics
  • drug delivery
  • biodistribution
  • pharmacokinetic

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Published Papers (1 paper)

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Research

25 pages, 7831 KiB  
Article
In Vitro Studies to Evaluate the Intestinal Permeation of an Ursodeoxycholic Acid-Conjugated Oligonucleotide for Duchenne Muscular Dystrophy Treatment
by Marika Faiella, Giada Botti, Alessandro Dalpiaz, Lorenzo Gnudi, Aurélie Goyenvalle, Barbara Pavan, Daniela Perrone, Matteo Bovolenta and Elena Marchesi
Pharmaceutics 2024, 16(8), 1023; https://doi.org/10.3390/pharmaceutics16081023 - 1 Aug 2024
Viewed by 1278
Abstract
Delivery represents a major hurdle to the clinical advancement of oligonucleotide therapeutics for the treatment of disorders such as Duchenne muscular dystrophy (DMD). In this preliminary study, we explored the ability of 2′-O-methyl-phosphorothioate antisense oligonucleotides (ASOs) conjugated with lipophilic ursodeoxycholic acid [...] Read more.
Delivery represents a major hurdle to the clinical advancement of oligonucleotide therapeutics for the treatment of disorders such as Duchenne muscular dystrophy (DMD). In this preliminary study, we explored the ability of 2′-O-methyl-phosphorothioate antisense oligonucleotides (ASOs) conjugated with lipophilic ursodeoxycholic acid (UDCA) to permeate across intestinal barriers in vitro by a co-culture system of non-contacting IEC-6 cells and DMD myotubes, either alone or encapsulated in exosomes. UDCA was used to enhance the lipophilicity and membrane permeability of ASOs, potentially improving oral bioavailability. Exosomes were employed due to their biocompatibility and ability to deliver therapeutic cargo across biological barriers. Exon skipping was evaluated in the DMD myotubes to reveal the targeting efficiency. Exosomes extracted from milk and wild-type myotubes loaded with 5′-UDC-3′Cy3-ASO and seeded directly on DMD myotubes appear able to fuse to myotubes and induce exon skipping, up to ~20%. Permeation studies using the co-culture system were performed with 5′-UDC-3′Cy3-ASO 51 alone or loaded in milk-derived exosomes. In this setting, only gymnotic delivery induced significant levels of exon skipping (almost 30%) implying a possible role of the intestinal cells in enhancing delivery of ASOs. These results warrant further investigations to elucidate the delivery of ASOs by gymnosis or exosomes. Full article
(This article belongs to the Special Issue Delivery of Oligonucleotide Therapeutics)
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