ArticleMolecular therapy. Methods & clinical development2024
Optimal different adeno-associated virus capsid/promoter combinations to target specific cell types in the common marmoset cerebral cortex.
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 11 papers.
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Who cites it
11 citing papers in PubMed.
- A Comprehensive Study of AAV Tropism in the Marmoset Brain.Neuroscience bulletin · 2026Article
- Noninvasive Focal Gene Delivery into the Cerebellum of Non-Human Primates using Focused Ultrasound.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2026Article
- Safety of Adeno-Associated Viral Vectors in Gene Therapy: Mechanisms of Toxicity, Clinical Risks, and Strategies for Their Minimization.International journal of molecular sciences · 2026Review
- Comparison of AAV9-driven motor neuron transduction following different CNS-directed delivery methods in mice.Scientific reports · 2026Article
- Targeted gene transfer into developmentally defined cell populations of the primate brain.Cell reports · 2026Article
- Developmental trajectories of vocal behaviors in common marmosets as a reference framework for neurobehavioral studies.Frontiers in neural circuits · 2026Article
- Ubiquitous and Brain Cell-Type-Specific Promoters and Enhancers in Lentiviral Vectors.Methods in molecular biology (Clifton, N.J.) · 2026Article
- Stem cell and gene therapies for leukodystrophies.Molecular therapy. Methods & clinical development · 2025Review
- Blood-brain barrier-penetrant AAV vectors for cell type-specific gene expression in the mouse brain.Anatomical science international · 2025Review
- RNA-programmable cell-type monitoring and manipulation in the human cortex with CellREADR.Cell reports · 2025Article
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4 authors.
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Abstract
To achieve cell-type-specific gene expression, using target cell-type-tropic different adeno-associated virus (AAV) capsids is advantageous. However, their tropism across brain cell types in nonhuman primates has not been fully elucidated. We assessed the tropism of nine AAV serotype capsids (AAV1, 2, 5, 6, 7, 8, 9, rh10, and DJ) expressing EGFP by chicken β-actin hybrid (CBh) promoter in marmoset cerebral cortical cells. All nine AAV capsid vectors, especially AAV9 and AAVrh10, caused highly neuron-selective EGFP expression. Some AAV capsids, including AAV5, induced EGFP expression to a lesser extent in oligodendrocytes. Different ubiquitous cytomegalovirus (CMV) and CMV early enhancer/chicken β-actin (CAG) promoters exhibited similar neuron-predominant transgene expression. Conversely, all nine AAV capsid vectors with the astrocyte-specific hGFA(ABC1D) promoter selectively expressed EGFP in astrocytes, except AAV5, which modestly expressed EGFP in oligodendrocytes. Oligodendrocyte-specific mouse myelin basic protein (mMBP) promoter in AAV5 vectors expressed EGFP in oligodendrocytes specifically and efficiently. The following are optimal combinations of capsids and promoters for cell-type-specific expression: AAV9 or AAVrh10 and ubiquitous CBh or CMV promoter for neuron-specific transgene expression, AAV2 or AAV7 and hGFA(ABC1D) promoters for astrocyte-specific transgene expression, and AAV5 and mMBP promoters for oligodendrocyte-specific transgene expression.
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