Jonathan D Cooper
Professor
Pediatrics
Los Angeles Biomedical Research Institute
United States of America
Biography
Dr. Cooper established his Pediatric Storage Disorders Laboratory (PDSL) in 2000 after his postdoc training at UCSF and Stanford. He is a leading international authority on the neuropathology of Batten disease or Neuronal Ceroid Lipofuscinosis (NCLs), and other lysosomal storage disorders. By characterizing mouse and larger animal models of NCL, and human cases, his lab has made several key discoveries in these disorders including the selective nature of neuron loss, and the importance of early glial activation in rather than the accumulation of storage material these disorders. More recently, his work has extended to the effects of disease outside the brain including the spinal cord, peripheral and autonomic nervous systems, and other organs including the heart. Together with colleagues from around the world his lab is also involved in the pre-clinical testing of a range of therapeutic strategies including enzyme replacement, gene therapy, neural stem cell transplantation and small molecule drugs, several of which have reached clinical trials.
Research Interest
Enzyme replacement, gene therapy, neural stem cell transplantation and small molecule drugs
Publications
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Intrathecal enzyme replacement therapy improves motor function and survival in a preclinical mouse model of infantile neuronal ceroid lipofuscinosis. Molecular genetics and metabolism
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Nonclinical evaluation of CNS-administered TPP1 enzyme replacement in canine CLN2 neuronal ceroid lipofuscinosis. Mol Genet Metab
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Towards a new understanding of NCL pathogenesis. Biochimica et biophysica acta. 2015;
