Jonathan Cooper, PhD

For the last 20 years, work in Jonathan Cooper’s Pediatric Storage Disorders Laboratory (PSDL) has focused upon investigating the pathogenesis of the neuronal ceroid lipofuscinoses (NCLs, or Batten disease), and other similar neurodegenerative lysosomal storage disorders. They have unbiased stereology to characterize multiple mouse and larger animal models NCL, and have used this information in order to be able to target a range of different pre-clinical interventions to where they can be most effective. This work has led to several clinical trials and an approved treatment for CLN2 disease.