نتایج جستجو برای: lysosomal storage diseases

تعداد نتایج: 1035749  

Journal: :European Journal of Human Genetics 2003

Journal: :Therapeutic Advances in Endocrinology and Metabolism 2010

Journal: :Frontiers in Molecular Biosciences 2020

Journal: :Clinical chemistry 2013
Baoyun Xia Ghazia Asif Leonard Arthur Muhammad A Pervaiz Xueli Li Renpeng Liu Richard D Cummings Miao He

BACKGROUND There are 45 known genetic diseases that impair the lysosomal degradation of macromolecules. The loss of a single lysosomal hydrolase leads to the accumulation of its undegraded substrates in tissues and increases of related glycoconjugates in urine, some of which can be detected by screening of free oligosaccharides (FOS) in urine. Traditional 1-dimensional TLC for urine oligosaccha...

Journal: :Philosophical transactions of the Royal Society of London. Series B, Biological sciences 2003
Volkmar Gieselmann Ulrich Matzner Diana Klein Jan Eric Mansson Rudi D'Hooge Peter D DeDeyn Renate Lüllmann Rauch Dieter Hartmann Klaus Harzer

Lysosomal storage diseases comprise a group of about 40 disorders, which in most cases are due to the deficiency of a lysosomal enzyme. Since lysosomal enzymes are involved in the degradation of various compounds, the diseases can be further subdivided according to which pathway is affected. Thus, enzyme deficiencies in the degradation pathway of glycosaminoglycans cause mucopolysaccharidosis, ...

Journal: :Current opinion in pediatrics 2010
Toya Ohashi

Enzyme replacement therapy (ERT) has been approved for 6 lysosomal storage diseases (LSDs) worldwide including Japan. These diseases include Gaucher disease (GD), Fabry disease, mucopolysaccharidosis (MPS) types I, II, and VI, and Pompe disease (PD). The efficacy and safety of ERT for LSDs has been confirmed by extensive clinical trials. However, there are still obstacles to successful ERT, suc...

2013
Daisuke Tsuji Kohji Itoh

Lysosomes are organella involving the catabolism of biomolecules extracellularly and intra‐ cellularly incorporated, which contain more than 60 distinct acidic hydrolases (lysosomal enzymes) and their co-factors. Lysosomal storage diseases (LSDs) are caused by germ-line gene mutations encoding lysosomal enzymes, their activator proteins, integral membrane proteins, cholesterol transporters and ...

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