Literature DB >> 859064

Cholesterol ester storage disease: clinical, biochemical, and pathological studies.

A L Beaudet, G D Ferry, B L Nichols, H S Rosenberg.   

Abstract

Of three siblings affected with cholesterol ester storage disease, two died at ages 7 and 9 years, respectively, with hepatic scarring and portal hypertension. Lipid storage was documented in both patients, as were esophageal varices and aortic plaques in the older child. The third affected sibling, followed to 13 years of age, has hepatomegaly, hyperlipidemia, short stature, adrenal calcification, and acid lipase deficiency. Leukocyte extracts demonstrated deficiency of acid lipase in this patient. This autosomal recessive condition may be allelic with Wolman disease with a more malignant course in this family than in most reported cases.

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Year:  1977        PMID: 859064     DOI: 10.1016/s0022-3476(77)80557-x

Source DB:  PubMed          Journal:  J Pediatr        ISSN: 0022-3476            Impact factor:   4.406


  16 in total

1.  Long-term treatment of a homozygous cholesteryl ester storage disease with combined cholestyramine and lovastatin.

Authors:  S Yokoyama; E McCoy
Journal:  J Inherit Metab Dis       Date:  1992       Impact factor: 4.982

2.  Novel LIPA mutations in Mexican siblings with lysosomal acid lipase deficiency.

Authors:  Yuritzi Santillán-Hernández; Enory Almanza-Miranda; Winnie W Xin; Kendrick Goss; Aurea Vera-Loaiza; María T Gorráez-de la Mora; Raul E Piña-Aguilar
Journal:  World J Gastroenterol       Date:  2015-01-21       Impact factor: 5.742

3.  Acid lipase cross-reacting material in Wolman disease and cholesterol ester storage disease.

Authors:  B K Burton; S P Reed
Journal:  Am J Hum Genet       Date:  1981-03       Impact factor: 11.025

4.  Sebelipase alfa over 52 weeks reduces serum transaminases, liver volume and improves serum lipids in patients with lysosomal acid lipase deficiency.

Authors:  Vassili Valayannopoulos; Vera Malinova; Tomas Honzík; Manisha Balwani; Catherine Breen; Patrick B Deegan; Gregory M Enns; Simon A Jones; John P Kane; Eveline O Stock; Radhika Tripuraneni; Stephen Eckert; Eugene Schneider; Gavin Hamilton; Michael S Middleton; Claude Sirlin; Bruce Kessler; Christopher Bourdon; Simeon A Boyadjiev; Reena Sharma; Chris Twelves; Chester B Whitley; Anthony G Quinn
Journal:  J Hepatol       Date:  2014-06-30       Impact factor: 25.083

5.  Clinical effect and safety profile of recombinant human lysosomal acid lipase in patients with cholesteryl ester storage disease.

Authors:  Manisha Balwani; Catherine Breen; Gregory M Enns; Patrick B Deegan; Tomas Honzík; Simon Jones; John P Kane; Vera Malinova; Reena Sharma; Eveline O Stock; Vassili Valayannopoulos; J Edmond Wraith; Jennifer Burg; Stephen Eckert; Eugene Schneider; Anthony G Quinn
Journal:  Hepatology       Date:  2013-03-28       Impact factor: 17.425

6.  A novel transgenic mouse model of lysosomal storage disorder.

Authors:  Sonia Ortiz-Miranda; Rui Ji; Agata Jurczyk; Ken-Edwin Aryee; Shunyan Mo; Terry Fletcher; Scott A Shaffer; Dale L Greiner; Rita Bortell; Ronald G Gregg; Alan Cheng; Leah J Hennings; Ann R Rittenhouse
Journal:  Am J Physiol Gastrointest Liver Physiol       Date:  2016-09-22       Impact factor: 4.052

7.  Hepatocyte-Specific Expression of Human Lysosome Acid Lipase Corrects Liver Inflammation and Tumor Metastasis in lal(-/-) Mice.

Authors:  Hong Du; Ting Zhao; Xinchun Ding; Cong Yan
Journal:  Am J Pathol       Date:  2015-07-26       Impact factor: 4.307

8.  Cholesteryl ester storage disease and Wolman disease: phenotypic variants of lysosomal acid cholesteryl ester hydrolase deficiency.

Authors:  J M Hoeg; S J Demosky; O H Pescovitz; H B Brewer
Journal:  Am J Hum Genet       Date:  1984-11       Impact factor: 11.025

9.  Wolman disease/cholesteryl ester storage disease: efficacy of plant-produced human lysosomal acid lipase in mice.

Authors:  Hong Du; Terri L Cameron; Stephen J Garger; Gregory P Pogue; Lee A Hamm; Earl White; Kathleen M Hanley; Gregory A Grabowski
Journal:  J Lipid Res       Date:  2008-04-15       Impact factor: 5.922

10.  Plasma lipids and lipoproteins of a patient with cholesteryl ester storage disease.

Authors:  G M Kostner; B Hadorn; A Roscher; R Zechner
Journal:  J Inherit Metab Dis       Date:  1985       Impact factor: 4.982

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