biostudies-arrayexpressSantiago NegueruelaMus musculushttps://www.ebi.ac.uk/biostudies/studies/E-MTAB-15988We observed a beneficial effect on Abca4KO mice phenotype upon miR-181a/b sponge treatment. Then, we decided to perform single cell analysis to better understand the pathway associated with amelioration observed. Abca4KO mice were injected with miR-181a/b sponge in one eye and with a control vector in the controlateral. Subretinal injection were performed at 2 months of age and samples collected and analyzed at 4 months post-treatment. In a second experiment Abca4KO and WT mice were analyzed analyzed at 4 months.biostudies-arrayexpressSample Collection - Abca4KO mice were injected with miR-181a/b sponge in one eye and with a control vector in the controlateral. Subretinal injection were performed at 2 months of age and samples collected and analyzed at 4 months post-treatment.Sequencing - Sequencing of the libraries was conducted on a NovaSeq 6000 platform (Illumina), adhering to the manufacturer's instructions.Library Construction - cDNA and final libraries were constructed using the Single Cell 3′ Library and Gel Bead Kit V3.1 (10x Genomics). The quality of the cDNA was assessed with High Sensitivity D5000 ScreenTape using the Agilent 4200 TapeStation system (Agilent Technologies), while the final libraries were evaluated with High Sensitivity DNA D1000 ScreenTape (Agilent Technologies).Nucleic Acid Extraction - The cell suspension was loaded onto a Chromium Single Cell G Chip Kit (10x Genomics) and processed with the Chromium Single Cell Controller (10x Genomics) to produce single-cell gel bead emulsions, following the manufacturer's guidelines.OrganizationMINSEQE ScoreAssays and DataProcessed DataMAGE-TAB FilesData Transformation - Processed files correspond to the output filtered expression matrix from CellRangerMetabolomicsUnknownTranscriptomicsGenomicsProteomicsIllumina NovaSeq 6000RNA-seq of coding RNA from single cellsMus musculusUncovering mitochondrial defects in photoreceptors opens novel therapeutic opportunities for Stargardt diseaseSimona Brillante, Anna Diana, Mariagrazia Volpe, Marta Molinari, Eva Cipollaro, Santiago Negueruela, Elena Polishchuk, Erika Tenderini, Carla Damiano, Patrizia Tornabene, Roman Polishchuk, Giancarlo Parenti, Antonietta Tarallo, Sandro Banfi, Ivana Trapani, Sabrina Carrella, Alessia IndrieriSantiago NegueruelafalseGene expression profile from single cell level of retinal cells from optic cup of injected Abca4KO mice with miR-181a/b sponge therapeuticWe observed a beneficial effect on Abca4KO mice phenotype upon miR-181a/b sponge treatment. Then, we decided to perform single cell analysis to better understand the pathway associated with amelioration observed. Abca4KO mice were injected with miR-181a/b sponge in one eye and with a control vector in the controlateral. Subretinal injection were performed at 2 months of age and samples collected and analyzed at 4 months post-treatment. In a second experiment Abca4KO and WT mice were analyzed analyzed at 4 months.2026-02-20T00:00:00Z2026-05-27T14:02:27.244Z2025-11-06T11:27:25.907ZE-MTAB-15988ERP183695EFO_0002944EFO_0004170EFO_0005684EFO_0005518EFO_0003816EFO_0004184