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UID:69da30ec59875
DTSTAMP:20260411T073052
DTSTART:20181130T120000
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TRANSP:OPAQUE
DTEND:20181130T130000
URL:https://murmitoyen.com/events/vanille/udem/detail/845869-the-same-but-d
 ifferent-towards-therapies-for-the-neuronal-ceroid-lipofuscinoses
LOCATION:CHU Sainte-Justine\, 3175\, Chemin de la Côte-Sainte-Catherine\, 
 Montréal\, QC\, Canada\, H3T 1C5
SUMMARY:The same\, but different: towards therapies for the neuronal ceroid
  lipofuscinoses
DESCRIPTION:Conférence scientifique | Centre de recherche du CHU Sainte-Ju
 stine  Conférence de Jonathan Cooper\, PhD\, professeur de pédiatrie\, g
 énétique et médecine génomique\, Université de Washington à Saint-Lo
 uis.
\nRésumé:The neuronal ceroid lipofuscinoses (NCLs or Batten disease
 ) are a group of fatal inherited lysosomal storage disorders. We are worki
 ng to understand how the brain is affected in these disorders\, and have i
 dentified vulnerable neuron populations\, and the contribution of glia to 
 disease progression. We are also testing a range of experimental therapies
  in both mouse and larger animal models of disease\, including enzyme repl
 acement and gene therapy. Our work suggests that these disorders are not a
 s similar as previously thought\, but detailed information about each form
  of NCL allows us to target therapies to where they can be most effective.
 ​
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