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An AAV capsid increases transduction of striatum and a ChAT promoter allows selective cholinergic neuron transduction.

Mol Ther Methods Clin Dev · 2023 · PMC10285237 · PMID 37359416

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In addition to increased spread of transduction within the brain, we significantly increased AAV transduction selectivity and showed a trend toward greater efficiency of striatal cholinergic neurons in vivo by using the truncated human ChAT promoter. 16 The ChAT promoter showed 7-fold higher specificity of transduction of cholinergic neurons than the ubiquitous CAG promoter ( Figure 2 E) when using AAV9 capsid and a 3-fold increase the absolute number of transduced cholinergic neurons ( Figure 2 F).

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