A hopeful therapy for Niemann-Pick C diseases

被引：6

作者：

Erickson, Robert P. ^{[1
]}

Fiorenza, Maria Teresa ^{[2
,3
]}

机构：

[1] Univ Arizona, Dept Pediat, Sch Med, Tucson, AZ 85724 USA

[2] Sapienza Univ, Dept Psychol, Div Neurosci, Rome, Italy

[3] IRCCS Fdn Santa Lucia, Rome, Italy

来源：

LANCET | 2017年 / 390卷 / 10104期

关键词：

STORAGE DISORDER; MOUSE; CYCLODEXTRINS; CHOLESTEROL;

D O I：

10.1016/S0140-6736(17)31631-8

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

引用

页码：1720 / 1721

页数：3

共 12 条

[1] CONTINUOUS INTRATHECAL BACLOFEN INFUSION FOR SPASTICITY OF CEREBRAL ORIGIN [J].

ALBRIGHT, AL ;

BARRON, WB ;

FASICK, MP ;

POLINKO, P ;

JANOSKY, J .

JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION, 1993, 270 (20) :2475-2477

[2] Cyclodextrins in the treatment of a mouse model of Niemann-Pick C disease [J].

Camargo, F ;

Erickson, RP ;

Garver, WS ;

Hossain, GS ;

Carbone, PN ;

Heidenreich, RA ;

Blanchard, J .

LIFE SCIENCES, 2001, 70 (02) :131-142

[3]

Christian AE, 1997, J LIPID RES, V38, P2264

[4] Cyclodextrin overcomes the transport defect in nearly every organ of NPC1 mice leading to excretion of sequestered cholesterol as bile acid [J].

Liu, Benny ;

Ramirez, Charina M. ;

Miller, Anna M. ;

Repa, Joyce J. ;

Turley, Stephen D. ;

Dietschy, John M. .

JOURNAL OF LIPID RESEARCH, 2010, 51 (05) :933-944

[5] Pulmonary function and pathology in hydroxypropyl-beta-cyclodextin-treated and untreated Npc1-/- mice [J].

Muralidhar, Akshay ;

Borbon, Ivan A. ;

Esharif, Dyadin M. ;

Ke, Wangjing ;

Manacheril, Rinu ;

Daines, Michael ;

Erickson, Robert P. .

MOLECULAR GENETICS AND METABOLISM, 2011, 103 (02) :142-147

[6] A marked paucity of granule cells in the developing cerebellum of the Npc1-/- mouse is corrected by a single injection of hydroxypropyl-β-cyclodextrin [J].

Nusca, S. ;

Canterini, S. ;

Palladino, G. ;

Bruno, F. ;

Mangia, F. ;

Erickson, R. P. ;

Fiorenza, M. T. .

NEUROBIOLOGY OF DISEASE, 2014, 70 :117-126

[7]

Ory D. S., 2017, LANCET

[8]

PENTCHEV PG, 1986, J BIOL CHEM, V261, P2772

[9] PARENTERAL HYDROXYPROPYL CYCLODEXTRINS - INTRAVENOUS AND INTRACEREBRAL ADMINISTRATION OF LIPOPHILES [J].

PITHA, J ;

GERLOCZY, A ;

OLIVI, A .

JOURNAL OF PHARMACEUTICAL SCIENCES, 1994, 83 (06) :833-837

[10] Impaired Autophagy in the Lipid-Storage Disorder Niemann-Pick Type C1 Disease [J].

Sarkar, Sovan ;

Carroll, Bernadette ;

Buganim, Yosef ;

Maetzel, Dorothea ;

Ng, Alex H. M. ;

Cassady, John P. ;

Cohen, Malkiel A. ;

Chakraborty, Souvik ;

Wang, Haoyi ;

Spooner, Eric ;

Ploegh, Hidde ;

Gsponer, Joerg ;

Korolchuk, Viktor I. ;

Jaenisch, Rudolf .

CELL REPORTS, 2013, 5 (05) :1302-1315

← 1 2 →