Future perspectives: Moving towards NCL treatments

被引：7

作者：

Cotman, Susan L. ^{[1
,2
]}

Mole, Sara E. ^{[3
]}

Kohan, Romina ^{[4
]}

机构：

[1] Harvard Univ, Massachusetts Gen Hosp, Sch Med, Ctr Human Genet Res, Boston, MA 02114 USA

[2] Harvard Univ, Massachusetts Gen Hosp, Sch Med, Dept Neurol, Boston, MA 02114 USA

[3] UCL, UCL Inst Child Hlth, Dept Genet Evolut & Environm, MRC Lab Cell Biol, London WC1E 6BT, England

[4] Univ Nacl Cordoba, Fac Ciencias Med, Ctr Estudio Metabolopatias Congenitas CEMECO, RA-5014 Cordoba, Argentina

来源：

BIOCHIMICA ET BIOPHYSICA ACTA-MOLECULAR BASIS OF DISEASE | 2015年 / 1852卷 / 10期

关键词：

Neuronal Ceroid Lipofuscinosis (NCL); Batten disease; NEURONAL CEROID-LIPOFUSCINOSIS; ENZYME REPLACEMENT; BATTEN-DISEASE; METABOLOMICS; MUTATION; BRAIN; PATHOLOGY; MODEL; TPP1; CLN3;

D O I：

10.1016/j.bbadis.2015.04.001

中图分类号：

Q5 [生物化学]; Q7 [分子生物学];

学科分类号：

071010 ; 081704 ;

摘要：

Clinicians, basic researchers, representatives from pharma and families from around the world met in Cordoba, Argentina in October, 2014 to discuss recent research progress at the 14th International Congress on Neuronal Ceroid Lipofuscinoses (NCLs; Batten disease), a group of clinically overlapping fatal, inherited lysosomal disorders with primarily neurodegenerative symptoms. This brief review article will provide perspectives on the anticipated future directions of NCL basic and clinical research as we move towards improved diagnosis, care and treatment of NCL patients. This article is part of a Special Issue entitled: Current Research on the Neuronal Ceroid Lipofuscinoses (Batten Disease). (C) 2015 Elsevier B.V. All rights reserved.

引用

页码：2336 / 2338

页数：3

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