نتایج جستجو برای: lysosomal storage

تعداد نتایج: 196223  

Journal: :Journal of child neurology 1996
A Prasad E M Kaye J Alroy

In this report, we have summarized our 9-year experience of over 100 proven cases of lysosomal storage disease using electron microscopic evaluation of skin biopsies as a screening tool. The skin biopsy was very specific in establishing the diagnosis in only two disorders, namely neuronal ceroid lipofuscinosis and mucolipidosis IV. Although the biopsy was not diagnostic in other categories of s...

2011
Diego L. Medina Alessandro Fraldi Valentina Bouche Fabio Annunziata Gelsomina Mansueto Carmine Spampanato Claudia Puri Antonella Pignata Jose A. Martina Marco Sardiello Michela Palmieri Roman Polishchuk Rosa Puertollano Andrea Ballabio

Lysosomes are cellular organelles primarily involved in degradation and recycling processes. During lysosomal exocytosis, a Ca²⁺-regulated process, lysosomes are docked to the cell surface and fuse with the plasma membrane (PM), emptying their content outside the cell. This process has an important role in secretion and PM repair. Here we show that the transcription factor EB (TFEB) regulates l...

Journal: :JAMA 1999
P J Meikle J J Hopwood A E Clague W F Carey

CONTEXT Lysosomal storage disorders represent a group of at least 41 genetically distinct, biochemically related, inherited diseases. Individually, these disorders are considered rare, although high prevalence values have been reported in some populations. These disorders are devastating for individuals and their families and result in considerable use of resources from health care systems; how...

Journal: :Pediatrics 2009
Orna Staretz-Chacham Tess C Lang Mary E LaMarca Donna Krasnewich Ellen Sidransky

Lysosomal storage disorders are rare inborn errors of metabolism, with a combined incidence of 1 in 1500 to 7000 live births. These relatively rare disorders are seldom considered when evaluating a sick newborn. A significant number of the >50 different lysosomal storage disorders, however, do manifest in the neonatal period and should be part of the differential diagnosis of several perinatal ...

Journal: :Nature Reviews Disease Primers 2018

Journal: :Nature Reviews Disease Primers 2019

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