Multifunctional Delivery Systems for Peptide Nucleic Acids

Volpi, Stefano; Cancelli, Umberto; Neri, Martina; Corradini, Roberto

doi:10.3390/ph14010014

Cited by 29 publications

(22 citation statements)

References 238 publications

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“…This strategy allows the uptake of the majority of the PNA molecules within few hours, reaching a maximum level (near 100%) after 72 h, as firstly published by Brognara et al [ 58 ] and then confirmed by several studies of our group [ 38 , 39 , 46 , 50 ]. The issue of PNA delivery has been fully addressed in a recent review paper by Volpi et al [ 59 ].…”

Section: Resultsmentioning

confidence: 99%

Combined Treatment of Bronchial Epithelial Calu-3 Cells with Peptide Nucleic Acids Targeting miR-145-5p and miR-101-3p: Synergistic Enhancement of the Expression of the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Gene

Papi

Gasparello

Zurlo

et al. 2022

IJMS

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The Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) gene encodes for a chloride channel defective in Cystic Fibrosis (CF). Accordingly, upregulation of its expression might be relevant for the development of therapeutic protocols for CF. MicroRNAs are deeply involved in the CFTR regulation and their targeting with miRNA inhibitors (including those based on Peptide Nucleic Acids, PNAs)is associated with CFTR upregulation. Targeting of miR-145-5p, miR-101-3p, and miR-335-5p with antisense PNAs was found to be associated with CFTR upregulation. The main objective of this study was to verify whether combined treatments with the most active PNAs are associated with increased CFTR gene expression. The data obtained demonstrate that synergism of upregulation of CFTR production can be obtained by combined treatments of Calu-3 cells with antisense PNAs targeting CFTR-regulating microRNAs. In particular, highly effective combinations were found with PNAs targeting miR-145-5p and miR-101-3p. Content of mRNAs was analyzed by RT-qPCR, the CFTR production by Western blotting. Combined treatment with antagomiRNAs might lead to maximized upregulation of CFTR and should be considered in the development of protocols for CFTR activation in pathological conditions in which CFTR gene expression is lacking, such as Cystic Fibrosis.

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Section: Resultsmentioning

confidence: 99%

Combined Treatment of Bronchial Epithelial Calu-3 Cells with Peptide Nucleic Acids Targeting miR-145-5p and miR-101-3p: Synergistic Enhancement of the Expression of the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Gene

Papi

Gasparello

Zurlo

et al. 2022

IJMS

Self Cite

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“…While these delivery methods and related recent findings will not be discussed in detail in this review, the readers can refer to comprehensive overviews of these PNA delivery mechanisms elsewhere. [ 100,103 ]…”

Section: Delivery Of Pnamentioning

confidence: 99%

Peptide nucleic acids and their role in gene regulation and editing

2021

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The unique properties of peptide nucleic acid (PNA) makes it a desirable candidate to be used in therapeutic and biotechnological interventions. It has been broadly utilized for numerous applications, with a major focus in regulation of gene expression, and more recently in gene editing. While the classic PNA design has mainly been employed to date, chemical modifications of the PNA backbone and nucleobases provide an avenue to advance the technology further. This review aims to discuss the recent developments in PNA based gene manipulation techniques and the use of novel chemical modifications to improve the current state of PNA mediated gene targeting.

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“…Furthermore, the precise control of gene knockdown provided by siRNA has allowed researchers to delineate important relationships between EWS/FLI and downstream targets such as MYC, IGFBP3, NR0B1, STEAP1, CALCB, and several neural crest specific genes [72] , [75] , [76] , [77] , [78] , [79] . The creation of nanoparticles containing these targeted oligonucleotides remains an ongoing area of investigation [80] , [81] . In addition, one such construct is currently being evaluated in the clinic (NCT02736565).…”

Section: Oligonucleotide Based Approaches Targeting Ews/flimentioning

confidence: 99%

One oncogene, several vulnerabilities: EWS/FLI targeted therapies for Ewing sarcoma

Flores

Grohar

2021

Journal of Bone Oncology

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Highlights EWS/FLI is the driver oncogene of Ewing sarcoma. EWS-FLI driven transcription yields numerous therapeutic vulnerabilities. The intrinsic cellular stress of dysregulated transcription leads to additional therapeutic vulnerabilities. A number of compounds targeting various steps in EWS/FLI driven transcription are in various stages of clinical development including those that are now in the clinic. The future of EWS/FLI targeting will likely focus on molecularly targeted combination therapies.

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Multifunctional Delivery Systems for Peptide Nucleic Acids

Cited by 29 publications

References 238 publications

Combined Treatment of Bronchial Epithelial Calu-3 Cells with Peptide Nucleic Acids Targeting miR-145-5p and miR-101-3p: Synergistic Enhancement of the Expression of the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Gene

Combined Treatment of Bronchial Epithelial Calu-3 Cells with Peptide Nucleic Acids Targeting miR-145-5p and miR-101-3p: Synergistic Enhancement of the Expression of the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Gene

Peptide nucleic acids and their role in gene regulation and editing

One oncogene, several vulnerabilities: EWS/FLI targeted therapies for Ewing sarcoma

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