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Glial Promoter Selectivity following AAV-Delivery to the Immature Brain
TLDR
The data suggest that direct AAV infusion to the developing postnatal brain, utilising cellular promoters, results in targeted and long-term transgene expression in glia, relevant for disease modelling and gene therapy for the treatment of glial pathology.
Aspartoacylase-LacZ Knockin Mice: An Engineered Model of Canavan Disease
TLDR
The aspalacZ mouse is an accurate model of CD and an important tool to identify novel aspects of its complex pathology.