[HTML][HTML] Neuronal ceroid lipofuscinoses
A Jalanko, T Braulke - Biochimica et Biophysica Acta (BBA)-Molecular Cell …, 2009 - Elsevier
The neuronal ceroid lipofuscinoses (NCL) are severe neurodegenerative lysosomal storage
disorders of childhood, characterized by accumulation of autofluorescent ceroid …
disorders of childhood, characterized by accumulation of autofluorescent ceroid …
The translational benefits of sheep as large animal models of human neurological disorders
SJ Murray, NL Mitchell - Frontiers in Veterinary Science, 2022 - frontiersin.org
The past two decades have seen a considerable rise in the use of sheep to model human
neurological disorders. While each animal model has its merits, sheep have many …
neurological disorders. While each animal model has its merits, sheep have many …
Longitudinal in vivo monitoring of the CNS demonstrates the efficacy of gene therapy in a sheep model of CLN5 Batten disease
NL Mitchell, KN Russell, MP Wellby, HE Wicky… - Molecular Therapy, 2018 - cell.com
Neuronal ceroid lipofuscinoses (NCLs; Batten disease) are neurodegenerative lysosomal
storage diseases predominantly affecting children. Single administration of brain-directed …
storage diseases predominantly affecting children. Single administration of brain-directed …
[HTML][HTML] Towards a new understanding of NCL pathogenesis
JD Cooper, MA Tarczyluk, HR Nelvagal - Biochimica Et Biophysica Acta …, 2015 - Elsevier
Abstract The Neuronal Ceroid Lipofuscinoses (NCLs, Batten disease) are a group of
inherited neurodegenerative disorders that have been traditionally grouped together on the …
inherited neurodegenerative disorders that have been traditionally grouped together on the …
[HTML][HTML] Use of model organisms for the study of neuronal ceroid lipofuscinosis
Neuronal ceroid lipofuscinoses are a group of fatal progressive neurodegenerative diseases
predominantly affecting children. Identification of mutations that cause neuronal ceroid …
predominantly affecting children. Identification of mutations that cause neuronal ceroid …
Intravitreal gene therapy protects against retinal dysfunction and degeneration in sheep with CLN5 Batten disease
Neuronal ceroid lipofuscinoses (NCL; Batten disease) are a group of inherited
neurodegenerative diseases primarily affecting children. A common feature across most …
neurodegenerative diseases primarily affecting children. A common feature across most …
A One Base Pair Deletion in the Canine ATP13A2 Gene Causes Exon Skipping and Late-Onset Neuronal Ceroid Lipofuscinosis in the Tibetan Terrier
A Wöhlke, U Philipp, P Bock, A Beineke… - PLoS …, 2011 - journals.plos.org
Neuronal ceroid lipofuscinosis (NCL) is a progressive neurodegenerative disease
characterized by brain and retinal atrophy and the intracellular accumulation of …
characterized by brain and retinal atrophy and the intracellular accumulation of …
[HTML][HTML] Canine neuronal ceroid lipofuscinoses: Promising models for preclinical testing of therapeutic interventions
ML Katz, E Rustad, GO Robinson, REH Whiting… - Neurobiology of …, 2017 - Elsevier
The neuronal ceroid lipofuscinoses (NCLs) are devastating inherited progressive
neurodegenerative diseases, with most forms having a childhood onset of clinical signs. The …
neurodegenerative diseases, with most forms having a childhood onset of clinical signs. The …
A lysosomal enigma CLN5 and its significance in understanding neuronal ceroid lipofuscinosis
I Basak, HE Wicky, KO McDonald, JB Xu… - Cellular and Molecular …, 2021 - Springer
Abstract Neuronal Ceroid Lipofuscinosis (NCL), also known as Batten disease, is an
incurable childhood brain disease. The thirteen forms of NCL are caused by mutations in …
incurable childhood brain disease. The thirteen forms of NCL are caused by mutations in …
A mutation in canine PPT1 causes early onset neuronal ceroid lipofuscinosis in a Dachshund
DN Sanders, FH Farias, GS Johnson, V Chiang… - Molecular genetics and …, 2010 - Elsevier
The neuronal ceroid lipofuscinoses (NCLs) are lysosomal storage diseases characterized
by progressive neurodegeneration and accumulation of autofluorescent storage granules. A …
by progressive neurodegeneration and accumulation of autofluorescent storage granules. A …