Article
Nonclinical evaluation of CNS-administered TPP1 enzyme replacement in canine CLN2 neuronal ceroid lipofuscinosis.
Molecular genetics and metabolism - 1 Feb 2015
Vuillemenot Brian R, Kennedy Derek, Cooper Jonathan D, Wong Andrew M S, Sri Sarmi, Doeleman Thom, Katz Martin L, Coates Joan R, Johnson Gayle C, Reed Randall P, Adams Eric L, Butt Mark T, Musson Donald G, Henshaw Joshua, Keve Steve, Cahayag Rhea, Tsuruda Laurie S, O'Neill Charles A
Abstract excerpt
The CLN2 form of neuronal ceroid lipofuscinosis, a type of Batten disease, is a lysosomal storage disorder caused by a deficiency of the enzyme tripeptidyl peptidase-1 (TPP1). Patients exhibit progressive neurodegeneration and loss of motor, cognitive, and visual functions, leading to death by the early teenage years. TPP1-null Dachshunds recapitulate human CLN2 disease. To characterize the safety and...
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