نتایج جستجو برای: sachs disease

تعداد نتایج: 1492092  

Journal: :Human molecular genetics 2017
Alexander W M Hooper Javier F Alamilla Rosemarie E Venier Deda C Gillespie Suleiman A Igdoura

GM2 gangliosidoses are a group of lysosomal storage disorders which include Sandhoff disease and Tay-Sachs disease. Dysregulation of glutamate receptors has been recently postulated in the pathology of Sandhoff disease. Glutamate receptor association with neuronal pentraxins 1 and 2, and the neuronal pentraxin receptor facilitates receptor potentiation and synaptic shaping. In this study, we ha...

2012
Gary Mockli

In 1887, Bernard Sachs reported on the clinical features diagnostic signs and symptoms and pathology of a patient with a condition that would someday bear his name(l). It was several decades later before it was discovered that these patients suffered from a recessive genetic trait resulting in the accumulation of a specific ganglioside, GM2, due to the deficiency of the lysosomal enzyme hexosam...

2015
Raelia M Lew Leslie Burnett Anné L Proos Martin B Delatycki

Tay-Sachs disease (TSD) is a fatal, recessively inherited neurodegenerative condition of infancy and early childhood. Although rare in most other populations, the carrier frequency is one in 25 in Ashkenazi Jews. Australian high-school-based TSD preconception genetic screening programs aim to screen, educate, and optimize reproductive choice for participants. These programs have demonstrated hi...

2010
Volkan Seyrantepe Pablo Lema Aurore Caqueret Larbi Dridi Samar Bel Hadj Stephane Carpentier Francine Boucher Thierry Levade Lionel Carmant Roy A. Gravel Edith Hamel Pascal Vachon Graziella Di Cristo Jacques L. Michaud Carlos R. Morales Alexey V. Pshezhetsky

Tay-Sachs disease is a severe lysosomal disorder caused by mutations in the HexA gene coding for the α-subunit of lysosomal β-hexosaminidase A, which converts G(M2) to G(M3) ganglioside. Hexa(-/-) mice, depleted of β-hexosaminidase A, remain asymptomatic to 1 year of age, because they catabolise G(M2) ganglioside via a lysosomal sialidase into glycolipid G(A2), which is further processed by β-h...

Journal: :Journal of Korean Medical Science 1993
H. W. Yoo K. H. Astrin R. J. Desnick

Tay-Sachs disease (GM2 gangliosidosis, type 1; TSD) is an autosomal recessive GM2 gangliosidosis resulting from the deficient activity of the lysosomal hydrolase beta-hexosaminidase A (Hex A). With a carrier frequency estimated at 1 in 25, it is a common lysosomal disorder in the Ashkenazi Jewish population. Tay-Sachs disease has provided the prototype for the prevention of severe recessive gen...

2003

Tay-Sachs disease (TSD) is a lysosomal storage disease that is inherited in an autosomal recessive pattern. Lysosomal storage diseases are a group of disorders characterized by deficiency of a specific single lysosomal enzyme, resulting in accumulation of abnormal metabolic products. TSD is characterized by a deficiency in a common lysosomal acid hydrolase, hexosaminidase A (Hex A). An insuffic...

Journal: :Obstetrics and gynecology 2009

Certain autosomal recessive disease conditions are more prevalent in individuals of Eastern European Jewish (Ashkenazi) descent. Previously, the American College of Obstetricians and Gynecologists recommended that individuals of Eastern European Jewish ancestry be offered carrier screening for Tay-Sachs disease, Canavan disease, and cystic fibrosis as part of routine obstetric care. Based on th...

2011
Kazuhiko Matsuoka Tomomi Tamura Daisuke Tsuji Yukie Dohzono Keisuke Kitakaze Kazuki Ohno Seiji Saito Hitoshi Sakuraba Kohji Itoh

To develop a novel enzyme replacement therapy for neurodegenerative Tay-Sachs disease (TSD) and Sandhoff disease (SD), which are caused by deficiency of β-hexosaminidase (Hex) A, we designed a genetically engineered HEXB encoding the chimeric human β-subunit containing partial amino acid sequence of the α-subunit by structure-based homology modeling. We succeeded in producing the modified HexB ...

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