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Compact Calm1 promoter enables AAV mediated neuron-targeted expression in human iPSC-derived brain organoids
Compact Calm1 promoter allows virus-based gene expression targeting neurons in human stem cell–derived brain models
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Abstract
The compact Calm1 promoter shows comparable performance to the widely used hSyn promoter in human neurons.
- The Calm1 promoter is 120 bp long, making it a smaller alternative for gene expression in the brain.
- Both the hSyn and Calm1 promoters demonstrated excitatory and inhibitory neuron-specificity in animal models.
- Gene expression was evaluated using reporter constructs in human -derived brain organoids.
- Findings indicate potential for the Calm1 promoter in central nervous system-targeted gene therapies.
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Key numbers
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Fluorescence Intensity Comparison
Fluorescence intensity of Calm1 vs. in human neurons
87 days
Organoid Age at Transduction
Age of organoids at the time of transduction