RNA Editing Therapeutics: Advances, Challenges and Perspectives on Combating Heart Disease

Sachse, Marco; Gatsiou, Aikaterini

doi:10.1007/s10557-022-07391-3

Cited by 11 publications

(6 citation statements)

References 128 publications

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“…miRbase (available at the following link , accessed on 4 February 2023) is a searchable database of published miRNA sequences and annotations; the current version used for this paper is 22.1, based on 38,589 entries [ 87 ]. For each miRNA entry, the miRBase database predicted a sequence database, representing a hairpin portion of a miRNA transcript, giving information on the location and sequence of the mature miRNA sequence (termed miR).…”

Section: Methodsmentioning

confidence: 99%

See 1 more Smart Citation

New Insight into Mechanisms of Cardiovascular Diseases: An Integrative Analysis Approach to Identify TheranoMiRNAs

Sessa

Salerno

Esposito

et al. 2023

IJMS

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MiRNAs regulate both physiological and pathological heart functions. Altered expression of miRNAs is associated with cardiovascular diseases (CVDs), making miRNAs attractive therapeutic strategies for the diagnosis and treatment of heart diseases. A recent publication defined, for the first time, the term theranoMiRNA, meaning the miRNAs that may be used both for diagnosis and treatment. The use of in silico tools may be considered fundamental for these purposes, clarifying several molecular aspects, suggesting future directions for in vivo studies. This study aims to explore different bioinformatic tools in order to clarify miRNA interactions with candidate genes, demonstrating the need to use a computational approach when establishing the most probable associations between miRNAs and target genes. This study focused on the functions of miR-133a-3p, miR-21-5p, miR-499a-5p, miR-1-3p, and miR-126-3p, providing an up-to-date overview, and suggests future lines of research in the identification of theranoMiRNAs related to CVDs. Based on the results of the present study, we elucidated the molecular mechanisms that could be linked between miRNAs and CVDs, confirming that these miRNAs play an active role in the genesis and development of heart damage. Given that CVDs are the leading cause of death in the world, the identification of theranoMiRNAs is crucial, hence the need for a definition of in vivo studies in order to obtain further evidence in this challenging field of research.

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Section: Methodsmentioning

confidence: 99%

“…Moreover, given that this database contains a link with the top 100 articles retrieved from PubMed, we selected the top 5 articles in order of relevance, selecting among these those that investigated cardiac function. This function is specific to this tool and has been fully described by Kozomara et al [ 87 ].…”

Section: Methodsmentioning

confidence: 99%

New Insight into Mechanisms of Cardiovascular Diseases: An Integrative Analysis Approach to Identify TheranoMiRNAs

Sessa

Salerno

Esposito

et al. 2023

IJMS

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“…Methods to control sequence-specific changes in nucleic acids have become powerful tools in molecular biology and hold promise for the therapeutic correction of disease-causing mutations [ 4 , 137 ]. Targeting gene mutations or fine-tuning of protein function at the RNA level rather than the DNA level is particularly attractive because RNA editing is reversible and regulatable without permanently altering to the genome [ 138 ]. Given that ADARs directly manipulate RNA, there is a growing interest in utilizing ADARs or directing endogenous ADARs to specific adenosines present in mRNA to address disease-associated G to A mutations in the genome [ 13 , 139 ].…”

Section: Site-directed Rna Editingmentioning

confidence: 99%

RNA editing enzymes: structure, biological functions and applications

Zhang,

Zhu,

Gao

et al. 2024

Cell Biosci

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With the advancement of sequencing technologies and bioinformatics, over than 170 different RNA modifications have been identified. However, only a few of these modifications can lead to base pair changes, which are called RNA editing. RNA editing is a ubiquitous modification in mammalian transcriptomes and is an important co/posttranscriptional modification that plays a crucial role in various cellular processes. There are two main types of RNA editing events: adenosine to inosine (A-to-I) editing, catalyzed by ADARs on double-stranded RNA or ADATs on tRNA, and cytosine to uridine (C-to-U) editing catalyzed by APOBECs. This article provides an overview of the structure, function, and applications of RNA editing enzymes. We discuss the structural characteristics of three RNA editing enzyme families and their catalytic mechanisms in RNA editing. We also explain the biological role of RNA editing, particularly in innate immunity, cancer biogenesis, and antiviral activity. Additionally, this article describes RNA editing tools for manipulating RNA to correct disease-causing mutations, as well as the potential applications of RNA editing enzymes in the field of biotechnology and therapy.

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“…Indeed, the inosine is read as guanosine by the ribosome and the amino acid tryptophane is inserted in the newly synthesized polypeptide [ 12 , 16 ]. Moreover, the sequence-specific RNA editing offers a promising and safer approach for correcting CFTR stop mutations compared to DNA editing methods [ 17 , 18 ]. In fact, RNA editing modifies the RNA transcript of a gene rather than the underlying DNA sequence and any side effects would then be less severe and would not be permanent.…”

Section: Introductionmentioning

confidence: 99%

Site-Specific RNA Editing of Stop Mutations in the CFTR mRNA of Human Bronchial Cultured Cells

Chiavetta

Titoli

Barra

et al. 2023

IJMS

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It is reported that about 10% of cystic fibrosis (CF) patients worldwide have nonsense (stop) mutations in the CFTR gene, which cause the premature termination of CFTR protein synthesis, leading to a truncated and non-functional protein. To address this issue, we investigated the possibility of rescuing the CFTR nonsense mutation (UGA) by sequence-specific RNA editing in CFTR mutant CFF-16HBEge, W1282X, and G542X human bronchial cells. We used two different base editor tools that take advantage of ADAR enzymes (adenosine deaminase acting on RNA) to edit adenosine to inosine (A-to-I) within the mRNA: the REPAIRv2 (RNA Editing for Programmable A to I Replacement, version 2) and the minixABE (A to I Base Editor). Immunofluorescence experiments show that both approaches were able to recover the CFTR protein in the CFTR mutant cells. In addition, RT-qPCR confirmed the rescue of the CFTR full transcript. These findings suggest that site-specific RNA editing may efficiently correct the UGA premature stop codon in the CFTR transcript in CFF-16HBEge, W1282X, and G542X cells. Thus, this approach, which is safer than acting directly on the mutated DNA, opens up new therapeutic possibilities for CF patients with nonsense mutations.

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RNA Editing Therapeutics: Advances, Challenges and Perspectives on Combating Heart Disease

Cited by 11 publications

References 128 publications

New Insight into Mechanisms of Cardiovascular Diseases: An Integrative Analysis Approach to Identify TheranoMiRNAs

New Insight into Mechanisms of Cardiovascular Diseases: An Integrative Analysis Approach to Identify TheranoMiRNAs

RNA editing enzymes: structure, biological functions and applications

Site-Specific RNA Editing of Stop Mutations in the CFTR mRNA of Human Bronchial Cultured Cells

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