Results 91 to 100 of about 2,448 (132)

GABAergic interneurons contribute to the fatal seizure phenotype of CLN2 disease mice. [PDF]

open access: yesJCI Insight
Takahashi K   +13 more
europepmc   +1 more source

Enzyme Replacement Therapy for CLN2 Disease: MRI Volumetry Shows Significantly Slower Volume Loss Compared with a Natural History Cohort. [PDF]

open access: yesAJNR Am J Neuroradiol
Gaur P   +9 more
europepmc   +1 more source

Recombinant human tripeptidyl peptidase-1 infusion to the monkey CNS: Safety, pharmacokinetics, and distribution

Toxicology and Applied Pharmacology, 2014
CLN2 disease is caused by deficiency in tripeptidyl peptidase-1 (TPP1), leading to neurodegeneration and death. The safety, pharmacokinetics (PK), and CNS distribution of recombinant human TPP1 (rhTPP1) were characterized following a single intracerebroventricular (ICV) or intrathecal-lumbar (IT-L) infusion to cynomolgus monkeys. Animals received 0, 5,
Mark T Butt   +2 more
exaly   +4 more sources

The lysosomal aminopeptidase tripeptidyl peptidase 1 displays increased activity in malignant pancreatic cysts

Biological Chemistry, 2019
Abstract Incidental detection of pancreatic cysts has increased dramatically over the last decade, but risk stratification and clinical management remain a challenge. Mucinous cysts are precursor lesions to pancreatic cancer, however, the majority are indolent.
Sam L, Ivry   +8 more
openaire   +2 more sources

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