In vitro and ex vivo screening of microRNA combinations with enhanced cell penetrating peptides to stimulate intervertebral disc regeneration

Abstract Background Low back pain (LBP) is predominantly caused by degeneration of the intervertebral disc (IVD) and central nucleus pulposus (NP) region. Conservative treatments fail to restore disc function, motivating the exploration of nucleic acid therapies, such as the use of microRNAs (miRNAs...

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Main Authors: Tara Ní Néill, Marcos N. Barcellona, Niamh Wilson, Fergal J. O'Brien, James E. Dixon, Caroline M. Curtin, Conor T. Buckley
Format: Article
Language:English
Published: Wiley 2024-12-01
Series:JOR Spine
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Online Access:https://doi.org/10.1002/jsp2.1366
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author Tara Ní Néill
Marcos N. Barcellona
Niamh Wilson
Fergal J. O'Brien
James E. Dixon
Caroline M. Curtin
Conor T. Buckley
author_facet Tara Ní Néill
Marcos N. Barcellona
Niamh Wilson
Fergal J. O'Brien
James E. Dixon
Caroline M. Curtin
Conor T. Buckley
author_sort Tara Ní Néill
collection DOAJ
description Abstract Background Low back pain (LBP) is predominantly caused by degeneration of the intervertebral disc (IVD) and central nucleus pulposus (NP) region. Conservative treatments fail to restore disc function, motivating the exploration of nucleic acid therapies, such as the use of microRNAs (miRNAs). miRNAs have the potential to modulate expression of discogenic factors, while silencing the catabolic cascade associated with degeneration. To deliver these miRNAs, nonviral cell penetrating peptides (CPPs) are gaining favor given their low immunogenicity and strong targeting ability. Single miRNA therapies have been investigated for IVD repair, however dual miRNA delivery strategies have not been commonly examined and may augment regeneration. Materials and methods Transfection of four pro‐discogenic miRNAs (miRNA mimics:140‐5p; 149‐5p and inhibitors: 141‐3p; 221‐3p) and dual delivery of six miRNA pairings was performed using two CPPs, RALA and GET peptide (FLR), in primary rat NP monolayer culture, and in an ex vivo organ culture model of rat caudal discs. Protein expression of discogenic (aggrecan, collagen type II, and SOX9) and catabolic markers (ADAMTS5 and MMP13) were assessed. Results Monolayer investigations signified enhanced discogenic marker expression following dual miRNA delivery, signifying a synergistic effect when compared to single miRNA transfection. Utilization of an appropriate model was emphasized in our ex vivo organ culture experiment, revealing the establishment of a regenerative microenvironment characterized by reduced catabolic enzyme activity and enhanced matrix deposition, particularly following concurrent delivery of FLR‐miRNA‐149‐5p mimic and miRNA‐221‐3p inhibitor. Bioinformatics analysis of miRNA‐149‐5p mimic and miRNA‐221‐3p inhibitor identified distinct targets, pathways, and interactions, suggesting a mode of action for this amplified response. Conclusion Our findings suggest the potential of FLR‐miRNA‐149‐5p + miRNA‐221‐3p inhibitor to create an anti‐catabolic niche within the disc to foster regeneration in moderate cases of disc degeneration, which could be utilized in further studies with the overarching aim of developing treatments for LBP.
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spelling doaj-art-d9e4e24821bf4c0fbb4262e3a0d23d352024-12-26T11:06:35ZengWileyJOR Spine2572-11432024-12-0174n/an/a10.1002/jsp2.1366In vitro and ex vivo screening of microRNA combinations with enhanced cell penetrating peptides to stimulate intervertebral disc regenerationTara Ní Néill0Marcos N. Barcellona1Niamh Wilson2Fergal J. O'Brien3James E. Dixon4Caroline M. Curtin5Conor T. Buckley6Trinity Centre for Biomedical Engineering Trinity Biomedical Sciences Institute, Trinity College Dublin, The University of Dublin Dublin IrelandTrinity Centre for Biomedical Engineering Trinity Biomedical Sciences Institute, Trinity College Dublin, The University of Dublin Dublin IrelandTrinity Centre for Biomedical Engineering Trinity Biomedical Sciences Institute, Trinity College Dublin, The University of Dublin Dublin IrelandTrinity Centre for Biomedical Engineering Trinity Biomedical Sciences Institute, Trinity College Dublin, The University of Dublin Dublin IrelandRegenerative Medicine and Cellular Therapies The University of Nottingham Biodiscovery Institute (BDI), School of Pharmacy, University of Nottingham Nottingham UKTrinity Centre for Biomedical Engineering Trinity Biomedical Sciences Institute, Trinity College Dublin, The University of Dublin Dublin IrelandTrinity Centre for Biomedical Engineering Trinity Biomedical Sciences Institute, Trinity College Dublin, The University of Dublin Dublin IrelandAbstract Background Low back pain (LBP) is predominantly caused by degeneration of the intervertebral disc (IVD) and central nucleus pulposus (NP) region. Conservative treatments fail to restore disc function, motivating the exploration of nucleic acid therapies, such as the use of microRNAs (miRNAs). miRNAs have the potential to modulate expression of discogenic factors, while silencing the catabolic cascade associated with degeneration. To deliver these miRNAs, nonviral cell penetrating peptides (CPPs) are gaining favor given their low immunogenicity and strong targeting ability. Single miRNA therapies have been investigated for IVD repair, however dual miRNA delivery strategies have not been commonly examined and may augment regeneration. Materials and methods Transfection of four pro‐discogenic miRNAs (miRNA mimics:140‐5p; 149‐5p and inhibitors: 141‐3p; 221‐3p) and dual delivery of six miRNA pairings was performed using two CPPs, RALA and GET peptide (FLR), in primary rat NP monolayer culture, and in an ex vivo organ culture model of rat caudal discs. Protein expression of discogenic (aggrecan, collagen type II, and SOX9) and catabolic markers (ADAMTS5 and MMP13) were assessed. Results Monolayer investigations signified enhanced discogenic marker expression following dual miRNA delivery, signifying a synergistic effect when compared to single miRNA transfection. Utilization of an appropriate model was emphasized in our ex vivo organ culture experiment, revealing the establishment of a regenerative microenvironment characterized by reduced catabolic enzyme activity and enhanced matrix deposition, particularly following concurrent delivery of FLR‐miRNA‐149‐5p mimic and miRNA‐221‐3p inhibitor. Bioinformatics analysis of miRNA‐149‐5p mimic and miRNA‐221‐3p inhibitor identified distinct targets, pathways, and interactions, suggesting a mode of action for this amplified response. Conclusion Our findings suggest the potential of FLR‐miRNA‐149‐5p + miRNA‐221‐3p inhibitor to create an anti‐catabolic niche within the disc to foster regeneration in moderate cases of disc degeneration, which could be utilized in further studies with the overarching aim of developing treatments for LBP.https://doi.org/10.1002/jsp2.1366anti‐catabolismcell penetrating peptidescell therapiesmicroRNAregeneration
spellingShingle Tara Ní Néill
Marcos N. Barcellona
Niamh Wilson
Fergal J. O'Brien
James E. Dixon
Caroline M. Curtin
Conor T. Buckley
In vitro and ex vivo screening of microRNA combinations with enhanced cell penetrating peptides to stimulate intervertebral disc regeneration
JOR Spine
anti‐catabolism
cell penetrating peptides
cell therapies
microRNA
regeneration
title In vitro and ex vivo screening of microRNA combinations with enhanced cell penetrating peptides to stimulate intervertebral disc regeneration
title_full In vitro and ex vivo screening of microRNA combinations with enhanced cell penetrating peptides to stimulate intervertebral disc regeneration
title_fullStr In vitro and ex vivo screening of microRNA combinations with enhanced cell penetrating peptides to stimulate intervertebral disc regeneration
title_full_unstemmed In vitro and ex vivo screening of microRNA combinations with enhanced cell penetrating peptides to stimulate intervertebral disc regeneration
title_short In vitro and ex vivo screening of microRNA combinations with enhanced cell penetrating peptides to stimulate intervertebral disc regeneration
title_sort in vitro and ex vivo screening of microrna combinations with enhanced cell penetrating peptides to stimulate intervertebral disc regeneration
topic anti‐catabolism
cell penetrating peptides
cell therapies
microRNA
regeneration
url https://doi.org/10.1002/jsp2.1366
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