[DKO study] Rescue of TDP-43 and neuronal pathologies by peripheral AAV-mediated expression of brain-penetrant progranulin in a mouse model of GRN-linked frontotemporal lobar degeneration Version 1
External LinkWe generated AAV(L):bPGRN (L = liver, b = brain-penetrant), a liver-targeting adenovirus (AAV) encoding a fusion protein (8D3:PGRN) consisting of a single chain fragment variable (scFv) antibody recognizing mouse TfR (transferrin receptor) fused to human PGRN (hPGRN). To study the effects of AAV(L):bPGRN, we treated 6-week old wildtype or GRN,TMEM106B double knock-out mice with either 1.3 x 10^13 vg/kg AAV or isotonic saline solution (0.9% NaCl) intravenously via tail vain injection. 9-10 weeks after the treatment the animals were sacrificed; they were anesthetized and transcardiacally perfused with PBS. From each animal, one hemibrain (excluding the olfactory bulb, but including cerebellum) was collected, snap frozen on dry ice and pulverized with a pistill. All animal experiments were performed according to German animal welfare law and approved by the government of upper Bavaria.
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Created: 2nd Dec 2024 at 09:53
Last updated: 2nd Dec 2024 at 09:58
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Version 1 (earliest) Created 2nd Dec 2024 at 09:53 by Aditi Methi
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Projects: Published Datasets, Unpublished Datasets
Institutions: LMU Klinikum
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Submitter: Rainer Malik
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Assays: Expression profiling: Bulk RNA-seq (human), Expression profiling: Bulk RNA-seq (human), Expression profiling: Bulk RNA-seq (human), Expression profiling: Bulk RNA-seq (human) + smallRNA-seq (human), Expression profiling: Bulk RNA-seq (mouse), Expression profiling: Bulk RNA-seq (mouse), Expression profiling: Bulk RNA-seq (mouse), Expression profiling: Bulk RNA-seq (mouse), Expression profiling: Bulk RNA-seq (mouse), Expression profiling: Bulk RNA-seq (mouse), Expression profiling: Bulk RNA-seq (mouse), Expression profiling: Bulk RNA-seq (mouse), Expression profiling: Bulk RNA-seq (mouse), Expression profiling: Bulk RNA-seq (mouse), Expression profiling: Bulk RNA-seq (mouse), Expression profiling: Bulk RNA-seq (rat), Expression profiling: MERFISH Spatial Transcriptomics (mouse), Expression profiling: Microarray (zebrafish), Expression profiling: RiboTag-mRNA-seq (mouse), Expression profiling: Small RNA-seq (human), Expression profiling: Spatial Transcriptomics (mouse), Expression profiling: Spatial Transcriptomics correlated Electron Micros..., Expression profiling: scRNA-seq (human), Expression profiling: scRNA-seq (human), Expression profiling: scRNA-seq (human), Expression profiling: scRNA-seq (human), Expression profiling: scRNA-seq (human) (Day 20), Expression profiling: scRNA-seq (human) (Day 5), Expression profiling: scRNA-seq (mouse), Expression profiling: scRNA-seq (mouse), Expression profiling: scRNA-seq (mouse), Expression profiling: scRNA-seq (mouse), Expression profiling: scRNA-seq (mouse), Expression profiling: scRNA-seq (mouse), Expression profiling: scRNA-seq (mouse), Expression profiling: scRNA-seq (mouse), Expression profiling: scRNA-seq (mouse), Expression profiling: scRNA-seq (mouse), Expression profiling: scRNA-seq (mouse), Expression profiling: scRNA-seq (mouse), Expression profiling: scRNA-seq (mouse), Expression profiling: scRNA-seq (mouse) + Bulk RNA-seq (mouse), Expression profiling: small RNA-seq (human), Expression profiling: small RNA-seq (mouse), Genome binding/occupancy profiling: Bulk ATAC-seq (mouse), Genome binding/occupancy profiling: Bulk ATAC-seq (mouse), Genome binding/occupancy profiling: CUT&Tag sequencing (mouse), Genome binding/occupancy profiling: snATAC-seq (mouse), Genome wide (GW) and validation CRISPR screens (rat)
Progranulin (PGRN) haploinsufficiency is a major risk factor for frontotemporal lobar degeneration with TAR DNA-binding protein 43 (TDP-43) pathology (FTLD-GRN). Multiple therapeutic strategies are in clinical development to restore PGRN in the CNS, including gene therapy. However, a limitation of current gene therapy approaches aimed to alleviate FTLD-associated pathologies may be their inefficient brain exposure and biodistribution. We therefore developed an adeno-associated virus (AAV) targeting ...
Submitter: Aditi Methi
Investigation: Transcriptomics (Published)
Submitter: Aditi Methi
Assay type: Transcriptomics
Technology type: Sequencing
Investigation: Transcriptomics (Published)
