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Advances in Nucleic Acid Therapeutics
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Advances in Nucleic Acid Therapeutics

A special issue of Life (ISSN 2075-1729). This special issue belongs to the section "Medical Research".

Deadline for manuscript submissions: closed (31 December 2022) | Viewed by 12890

Special Issue Editor

Dr. Wen Zhang

E-Mail Website
Guest Editor
Department of Biochemistry and Molecular Biology, Indiana University Melvin and Bren Simon Comprehensive Cancer Center, Indiana University School of Medicine, 635 Barnhill Drive, Indianapolis, IN 46202, USA
Interests: nucleic acids; structural characterization; therapeutics development; drug delivery

Special Issue Information

Dear Colleagues,

Nucleic-acid-based therapeutics have been recognized as a significant category of drug, in addition to traditional small molecules and antibody drugs. There has been tremendous progress in recent decades to utilize nucleic-acid-related molecules to treat a wide spectrum of diseases, among which several nucleic acid drugs have been approved for clinical use. Particularly in the past two years, we have witnessed the unprecedented global COVID-19 pandemic and the successful development of the first mRNA vaccine, which demonstrates the prominent potential of nucleic acid therapies. Moreover, the development of innovative chemical approaches and biological technologies make the exploration of nucleic acid drugs progress so rapidly that we think it is necessary to summarize some important developments in this field in the form of this Special Issue.

This Special Issue, “Advances in Nucleic Acid Therapeutics”, edited by Prof. Wen Zhang, will be devoted to the applications of different nucleic acid therapeutics, as well as their insights into the mechanisms. The topics will include nucleoside molecules, siRNA, protein-binding RNA, CRISPR gene editing, ribosomes, aptamers, ribozymes, and nanostructures.

This Special Issue is now open for submissions. Prospective authors should first send a short abstract or tentative title to the Editorial Office. If the editors deem the topic to be appropriate for inclusion in the Special Issue, the author will be encouraged to submit the full manuscript.

Dr. Wen Zhang
Guest Editor

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. Life 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 2600 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

  • nucleic acid
  • aptamer
  • siRNA
  • nucleoside
  • nanoparticle
  • ribosome
  • CRISPR

Published Papers (3 papers)

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Research

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10 pages, 5083 KiB  
Article
Selective RNA Labeling by RNA-Compatible Type II Restriction Endonuclease and RNA-Extending DNA Polymerase
by Hyesung Jo, Jiyun Beon and Seung Soo Oh
Life 2022, 12(10), 1674; https://doi.org/10.3390/life12101674 - 21 Oct 2022
Viewed by 3569
Abstract
RNAs not only offer valuable information regarding our bodies but also regulate cellular functions, allowing for their specific manipulations to be extensively explored for many different biological and clinical applications. In particular, rather than temporary hybridization, permanent labeling is often required to introduce [...] Read more.
RNAs not only offer valuable information regarding our bodies but also regulate cellular functions, allowing for their specific manipulations to be extensively explored for many different biological and clinical applications. In particular, rather than temporary hybridization, permanent labeling is often required to introduce functional tags to target RNAs; however, direct RNA labeling has been revealed to be challenging, as native RNAs possess unmodifiable chemical moieties or indefinable dummy sequences at the ends of their strands. In this work, we demonstrate the combinatorial use of RNA-compatible restriction endonucleases (REs) and RNA-extending polymerases for sequence-specific RNA cleavage and subsequent RNA functionalization. Upon the introduction of complementary DNAs to target RNAs, Type II REs, such as AvrII and AvaII, could precisely cut the recognition site in the RNA-DNA heteroduplexes with exceptionally high efficiency. Subsequently, the 3′ ends of the cleaved RNAs were selectively and effectively modified when Therminator DNA polymerase template-dependently extended the RNA primers with a variety of modified nucleotides. Based on this two-step RNA labeling, only the target RNA could be chemically labeled with the desired moieties, such as bioconjugation tags or fluorophores, even in a mixture of various RNAs, demonstrating the potential for efficient and direct RNA modifications. Full article
(This article belongs to the Special Issue Advances in Nucleic Acid Therapeutics)
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Review

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27 pages, 1204 KiB  
Review
CRISPR-Based Tools for Fighting Rare Diseases
by Qingyang Li, Yanmin Gao and Haifeng Wang
Life 2022, 12(12), 1968; https://doi.org/10.3390/life12121968 - 24 Nov 2022
Cited by 2 | Viewed by 4317
Abstract
Rare diseases affect the life of a tremendous number of people globally. The CRISPR-Cas system emerged as a powerful genome engineering tool and has facilitated the comprehension of the mechanism and development of therapies for rare diseases. This review focuses on current efforts [...] Read more.
Rare diseases affect the life of a tremendous number of people globally. The CRISPR-Cas system emerged as a powerful genome engineering tool and has facilitated the comprehension of the mechanism and development of therapies for rare diseases. This review focuses on current efforts to develop the CRISPR-based toolbox for various rare disease therapy applications and compares the pros and cons of different tools and delivery methods. We further discuss the therapeutic applications of CRISPR-based tools for fighting different rare diseases. Full article
(This article belongs to the Special Issue Advances in Nucleic Acid Therapeutics)
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16 pages, 1036 KiB  
Review
The Delivery of mRNA Vaccines for Therapeutics
by Nitika, Jiao Wei and Ai-Min Hui
Life 2022, 12(8), 1254; https://doi.org/10.3390/life12081254 - 17 Aug 2022
Cited by 29 | Viewed by 4275
Abstract
mRNA vaccines have been revolutionary in combating the COVID-19 pandemic in the past two years. They have also become a versatile tool for the prevention of infectious diseases and treatment of cancers. For effective vaccination, mRNA formulation, delivery method and composition of the [...] Read more.
mRNA vaccines have been revolutionary in combating the COVID-19 pandemic in the past two years. They have also become a versatile tool for the prevention of infectious diseases and treatment of cancers. For effective vaccination, mRNA formulation, delivery method and composition of the mRNA carrier play an important role. mRNA vaccines can be delivered using lipid nanoparticles, polymers, peptides or naked mRNA. The vaccine efficacy is influenced by the appropriate delivery materials, formulation methods and selection of a proper administration route. In addition, co-delivery of several mRNAs could also be beneficial and enhance immunity against various variants of an infectious pathogen or several pathogens altogether. Here, we review the recent progress in the delivery methods, modes of delivery and patentable mRNA vaccine technologies. Full article
(This article belongs to the Special Issue Advances in Nucleic Acid Therapeutics)
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