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September 02, 2020
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Spatial Profiling to Assess a Novel Gene Therapy in a Mouse Model of a Rare Genetic Disorder


Scientist II,
Ultragenyx Pharmaceutical

This webinar will discuss the use of digital spatial profiling to test a gene therapy for CDKL5 deficiency disorder (CDD), a rare X-linked neurodevelopmental disease that causes severe seizures, global developmental delay, and limits a patient’s ability to communicate.

Maggie Wright of Ultragenyx Pharmaceutical will outline her team’s approach to testing a gene therapy in a mouse model of CDD:

  • Adolescent CDKL5-/Y and CDKL5-/+ mice were administered the gene therapy AAV9-SYN-hCDKL5 via the intracerebral ventricular space
  • Distribution of the vector-derived hCDKL5 mRNA was assayed in the brain of these mice via RNAScope
  • Behavioral phenotypes of these treated mice were tested to determine efficacy of the treatment
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