نتایج جستجو برای: sandhoff disease

تعداد نتایج: 1490121  

Journal: :Human gene therapy 2016
Subha Karumuthil-Melethil Sahana Nagabhushan Kalburgi Patrick Thompson Michael Tropak Michael D Kaytor John G Keimel Brian L Mark Don Mahuran Jagdeep S Walia Steven J Gray

GM2 gangliosidosis is a family of three genetic neurodegenerative disorders caused by the accumulation of GM2 ganglioside (GM2) in neuronal tissue. Two of these are due to the deficiency of the heterodimeric (α-β), "A" isoenzyme of lysosomal β-hexosaminidase (HexA). Mutations in the α-subunit (encoded by HEXA) lead to Tay-Sachs disease (TSD), whereas mutations in the β-subunit (encoded by HEXB)...

2018
D Ito C Ishikawa N D Jeffery K Ono M Tsuboi K Uchida O Yamato M Kitagawa

A 13-month-old female Toy Poodle was presented for progressive ataxia and intention tremors of head movement. The diagnosis of Sandhoff's disease (GM2 gangliosidosis) was confirmed by deficient β-N-acetylhexosaminidase A and B activity in circulating leukocytes and identification of the homozygous mutation (HEXB: c.283delG). White matter in the cerebrum and cerebellum was hyperintense on T2-wei...

Journal: :Proceedings of The Japanese Society of Animal Models for Human Diseases 1996

2014
Sandra Merscher Alessia Fornoni

Sphingolipids are components of the lipid rafts in plasma membranes, which are important for proper function of podocytes, a key element of the glomerular filtration barrier. Research revealed an essential role of sphingolipids and sphingolipid metabolites in glomerular disorders of genetic and non-genetic origin. The discovery that glucocerebrosides accumulate in Gaucher disease in glomerular ...

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