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

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)...

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Veröffentlicht in:	Toxicology and applied pharmacology 2014-05, Vol.277 (1), p.49-57
Hauptverfasser:	Vuillemenot, Brian R., Kennedy, Derek, Reed, Randall P., Boyd, Robert B., Butt, Mark T., Musson, Donald G., Keve, Steve, Cahayag, Rhea, Tsuruda, Laurie S., O'Neill, Charles A.
Format:	Artikel
Sprache:	eng
Schlagworte:	60 APPLIED LIFE SCIENCES Aminopeptidases - administration & dosage Aminopeptidases - adverse effects Aminopeptidases - pharmacokinetics Aminopeptidases - therapeutic use ANIMAL TISSUES Animals Biological and medical sciences BLOOD CELLS CEREBROSPINAL FLUID Cerebrospinal Fluid - cytology CLN2 disease DEATH Dipeptidyl-Peptidases and Tripeptidyl-Peptidases - administration & dosage Dipeptidyl-Peptidases and Tripeptidyl-Peptidases - adverse effects Dipeptidyl-Peptidases and Tripeptidyl-Peptidases - pharmacokinetics Dipeptidyl-Peptidases and Tripeptidyl-Peptidases - therapeutic use Disease Models, Animal DISEASES DISTRIBUTION Dose-Response Relationship, Drug DRUGS Enzyme replacement therapy Enzyme Replacement Therapy - methods ENZYMES Errors of metabolism Female HALF-LIFE Haplorhini HEMATOXYLIN INFUSION Infusions, Intraventricular Injections, Spinal Intracerebroventricular Intrathecal Leukocyte Count Lipids (lysosomal enzyme disorders, storage diseases) Medical sciences Metabolic diseases MONKEYS Neuronal ceroid lipofuscinosis Neuronal Ceroid-Lipofuscinoses - drug therapy OVARIES Recombinant Proteins SAFETY Serine Proteases - administration & dosage Serine Proteases - adverse effects Serine Proteases - pharmacokinetics Serine Proteases - therapeutic use THALAMUS THERAPY Toxicology Tripeptidyl peptidase-1
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