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Spatial Profiling to Assess a Novel Gene Therapy in a Mouse Model of a Rare Genetic Disorder
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"Spatial Profiling to Assess a Novel Gene Therapy in a Mouse Model of a Rare Genetic Disorder"

This webinar discusses the use of digital spatial profiling to test a gene therapy for a rare neurodevelopmental disease that causes severe seizures, global developmental delay, and limits a patient’s ability to communicate.

Maggie Wright of Ultragenyx Pharmaceutical outlines her team’s approach to testing a gene therapy in a mouse model.

She discusses how the mice were administered the gene therapy and using the RNAscope ISH assay, assessed the distribution of the gene therapy mRNA to determine treatment efficacy.


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