N-butyldeoxynojirimycin delays motor deficits, cerebellar microgliosis, and Purkinje cell loss in a mouse model of mucolipidosis type IV

Mucolipidosis type IV (MLIV) is a lysosomal storage disease exhibiting progressive intellectual disability, motor impairment, and premature death. There is currently no cure or corrective treatment. The disease results from mutations in the gene encoding mucolipin-1, a transient receptor potential c...

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Ngā taipitopito rārangi puna kōrero
Ngā kaituhi matua:	Lauren C. Boudewyn, Jakub Sikora, Ladislav Kuchar, Jana Ledvinova, Yulia Grishchuk, Shirley L. Wang, Kostantin Dobrenis, Steven U. Walkley
Hōputu:	Artigo
Reo:	Inglês
I whakaputaina:	Elsevier 2017-09-01
Rangatū:	Neurobiology of Disease
Ngā marau:	Mucolipidosis type IV Miglustat Lysosomal storage disease Small molecule therapy Mucolipin-1 Purkinje cells
Urunga tuihono:	http://www.sciencedirect.com/science/article/pii/S0969996117301304
Ngā Tūtohu:	Tāpirihia he Tūtohu Kāore He Tūtohu, Me noho koe te mea tuatahi ki te tūtohu i tēnei pūkete!

N-butyldeoxynojirimycin delays motor deficits, cerebellar microgliosis, and Purkinje cell loss in a mouse model of mucolipidosis type IV

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