Intrathecal enzyme replacement therapy improves motor function and survival in a preclinical mouse model of infantile neuronal ceroid lipofuscinosis

Medicine & Life Sciences

• Ceroid lipofuscinosis, neuronal 1, infantile 100%
• palmitoyl-protein thioesterase 76%
• Enzyme Replacement Therapy 74%
• Neuronal Ceroid-Lipofuscinoses 40%
• Knockout Mice 38%
• human PPT1 protein 31%
• Enzymes 31%
• Spinal Cord 23%
• Lysosomal Storage Diseases 19%
• Clinical Trials 18%
• Motor Skills 16%
• Neuropathology 15%
• Traction 15%
• Blindness 14%
• Cerebrospinal Fluid 12%
• Dementia 11%
• Seizures 10%
• Central Nervous System 10%
• Animal Models 10%
• Therapeutics 9%
• Staining and Labeling 9%
• Neurons 8%
• Brain 6%

Chemical Compounds

• Intrathecal 97%
• Disorder 48%
• Cord 46%
• Intraventricular 32%
• Volume 10%