ArticleMolecular therapy. Methods & clinical development2024
Improving cell-specific recombination using AAV vectors in the murine CNS by capsid and expression cassette optimization.
Article in Molecular therapy. Methods & clinical development, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 13 papers.
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13 citing papers in PubMed.
- Generation of a New Immunodeficient Rat Model of Retinal Degeneration With LSL TdTomato Reporter and TdTomato-Pcp2 Expression.Translational vision science & technology · 2026Article
- Cell type-specific contextualisation of the human phenome: towards the systematic treatment of all rare diseases.Genome medicine · 2026Article
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- Defining a Midgestational Window forbioRxiv : the preprint server for biology · 2026Article
- Optogenetic Strategies for Motor Recovery After Ischemic Stroke: Mechanisms and Therapeutic Implications.Cellular and molecular neurobiology · 2026Review
- Generation of a new immunodeficient rat model of retinal degeneration with LSL TdTomato reporter and TdTomato-Pcp2 expression.bioRxiv : the preprint server for biology · 2026Article
- Ubiquitous and Brain Cell-Type-Specific Promoters and Enhancers in Lentiviral Vectors.Methods in molecular biology (Clifton, N.J.) · 2026Article
- Blood-brain barrier-penetrant AAV vectors for cell type-specific gene expression in the mouse brain.Anatomical science international · 2025Review
- Efficient gene delivery admitted by small metabolites specifically targeting astrocytes in the mouse brain.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Article
- Optimal different adeno-associated virus capsid/promoter combinations to target specific cell types in the common marmoset cerebral cortex.Molecular therapy. Methods & clinical development · 2024Article
- Article
- In vivo selection in non-human primates identifies AAV capsids for on-target CSF delivery to spinal cord.Molecular therapy : the journal of the American Society of Gene Therapy · 2024Article
- Production of Spinocerebellar Ataxia Type 3 Model Mice by Intravenous Injection of AAV-PHP.B Vectors.International journal of molecular sciences · 2024Article
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12 authors.
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Abstract
The production of cell-type- and age-specific genetically modified mice is a powerful approach for unraveling unknown gene functions. Here, we present a simple and timesaving method that enables adeno-associated virus (AAV)-mediated cell-type- and age-specific recombination in floxed mice. To achieve astrocyte-specific recombination in floxed Ai14 reporter mice, we intravenously injected blood-brain barrier-penetrating AAV-PHP.eB vectors expressing Cre recombinase (Cre) using the astrocyte-specific mouse glial fibrillary acidic protein (mGfaABC1D) promoter. However, we observed nonspecific neuron-predominant transduction despite the use of an astrocyte-specific promoter. We speculated that subtle but continuous Cre expression in nonastrocytic cells triggers recombination, and that excess production of Cre in astrocytes inhibits recombination by forming Cre-DNA aggregates. Here, we resolved this paradoxical event by dividing a single AAV into two mGfaABC1D-promoter-driven AAV vectors, one expressing codon-optimized flippase (FlpO) and another expressing flippase recognition target-flanked rapidly degrading Cre (dCre), together with switching the neuron-tropic PHP.eB capsid to astrocyte-tropic AAV-F. Moreover, we found that the FlpO-dCre system with a target cell-tropic capsid can also function in neuron-targeting recombination in floxed mice.
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