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.
Offered Free by: ACD
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