Saved in:
| Main Authors: | , , , , , , , , , , , |
|---|---|
| Format: | Artículo científico |
| Language: | en |
| Published: |
Molecular therapy. Nucleic acids
2025
|
| Online Access: | https://pubmed.ncbi.nlm.nih.gov/39877002/ |
| Tags: |
Add Tag
No Tags, Be the first to tag this record!
|
| _version_ | 1868266249906028544 |
|---|---|
| author | Petrogiannakis, Georgios Guadagnino, Irene Negueruela, Santiago Di Guida, Martina Marrocco, Elena Pizzo, Mariateresa Torella, Annalaura Zanobio, Mariateresa Karali, Marianthi Medina, Diego Luis Carrella, Sabrina Banfi, Sandro |
| author_facet | Petrogiannakis, Georgios Guadagnino, Irene Negueruela, Santiago Di Guida, Martina Marrocco, Elena Pizzo, Mariateresa Torella, Annalaura Zanobio, Mariateresa Karali, Marianthi Medina, Diego Luis Carrella, Sabrina Banfi, Sandro Petrogiannakis, Georgios Guadagnino, Irene Negueruela, Santiago Di Guida, Martina Marrocco, Elena Pizzo, Mariateresa Torella, Annalaura Zanobio, Mariateresa Karali, Marianthi Medina, Diego Luis Carrella, Sabrina Banfi, Sandro |
| collection | PubMed - marine biology |
| contents | high-content screening reveals miR-429 as a protective molecule in photoreceptor degeneration. Petrogiannakis, Georgios Guadagnino, Irene Negueruela, Santiago Di Guida, Martina Marrocco, Elena Pizzo, Mariateresa Torella, Annalaura Zanobio, Mariateresa Karali, Marianthi Medina, Diego Luis Carrella, Sabrina Banfi, Sandro Inherited retinal diseases (IRDs) are clinically and genetically heterogeneous disorders characterized by progressive photoreceptor degeneration and irreversible vision loss. MicroRNAs (miRNAs), a class of endogenous non-coding RNAs with post-transcriptional regulatory properties, are known to play a major role in retinal function, both in physiological and pathological conditions. Given their ability to simultaneously modulate multiple molecular pathways, miRNAs represent promising therapeutic tools for disorders with high genetic heterogeneity, such as IRDs. In the present study, we performed high-content imaging (HCI) screening to assess the impact of miRNA overexpression on a photoreceptor cell line undergoing light-induced degeneration. More than 1,200 miRNAs were assayed for putative protective effects in light-stressed 661W photoreceptor-like cells, and the top-performing miRNAs were further validated in independent assays. miR-429 showed the strongest cell-protective effect . Adeno-associated viral vector-mediated subretinal delivery of miR-429 in the IRD mouse model preserved electrophysiological responses and was associated with reduced inflammatory processes in the retina. We demonstrate that the HCI assay we devised is a reliable screening method to select candidate molecules for mutation-independent therapeutic approaches for retinal disorders. Moreover, our data indicate that miR-429 represents a potential therapeutic target against photoreceptor degeneration. |
| format | Artículo científico |
| id | pubmed_39877002 |
| institution | PubMed |
| language | en |
| publishDate | 2025 |
| publisher | Molecular therapy. Nucleic acids |
| record_format | pubmed |
| spellingShingle | high-content screening reveals miR-429 as a protective molecule in photoreceptor degeneration. Petrogiannakis, Georgios Guadagnino, Irene Negueruela, Santiago Di Guida, Martina Marrocco, Elena Pizzo, Mariateresa Torella, Annalaura Zanobio, Mariateresa Karali, Marianthi Medina, Diego Luis Carrella, Sabrina Banfi, Sandro high-content screening reveals miR-429 as a protective molecule in photoreceptor degeneration. Petrogiannakis, Georgios Guadagnino, Irene Negueruela, Santiago Di Guida, Martina Marrocco, Elena Pizzo, Mariateresa Torella, Annalaura Zanobio, Mariateresa Karali, Marianthi Medina, Diego Luis Carrella, Sabrina Banfi, Sandro Inherited retinal diseases (IRDs) are clinically and genetically heterogeneous disorders characterized by progressive photoreceptor degeneration and irreversible vision loss. MicroRNAs (miRNAs), a class of endogenous non-coding RNAs with post-transcriptional regulatory properties, are known to play a major role in retinal function, both in physiological and pathological conditions. Given their ability to simultaneously modulate multiple molecular pathways, miRNAs represent promising therapeutic tools for disorders with high genetic heterogeneity, such as IRDs. In the present study, we performed high-content imaging (HCI) screening to assess the impact of miRNA overexpression on a photoreceptor cell line undergoing light-induced degeneration. More than 1,200 miRNAs were assayed for putative protective effects in light-stressed 661W photoreceptor-like cells, and the top-performing miRNAs were further validated in independent assays. miR-429 showed the strongest cell-protective effect . Adeno-associated viral vector-mediated subretinal delivery of miR-429 in the IRD mouse model preserved electrophysiological responses and was associated with reduced inflammatory processes in the retina. We demonstrate that the HCI assay we devised is a reliable screening method to select candidate molecules for mutation-independent therapeutic approaches for retinal disorders. Moreover, our data indicate that miR-429 represents a potential therapeutic target against photoreceptor degeneration. |
| title | high-content screening reveals miR-429 as a protective molecule in photoreceptor degeneration. |
| url | https://pubmed.ncbi.nlm.nih.gov/39877002/ |