Literature DB >> 27867988

A canine model for neuronal ceroid lipofuscinosis highlights the promise of gene therapy for lysosomal storage diseases.

Jonathan E Phillips1, Richard H Gomer2.   

Abstract

Entities:  

Year:  2016        PMID： 27867988      PMCID： PMC5104632          DOI： 10.21037/atm.2016.10.16

Source DB:  PubMed          Journal:  Ann Transl Med        ISSN： 2305-5839


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  25 in total

Review 1.  Gene therapy for the neurological manifestations in lysosomal storage disorders.

Authors:  Seng H Cheng
Journal:  J Lipid Res       Date:  2014-03-29       Impact factor: 5.922

2.  Forebrain ependymal cells are Notch-dependent and generate neuroblasts and astrocytes after stroke.

Authors:  Marie Carlén; Konstantinos Meletis; Christian Göritz; Vladimer Darsalia; Emma Evergren; Kenji Tanigaki; Mario Amendola; Fanie Barnabé-Heider; Maggie S Y Yeung; Luigi Naldini; Tasuku Honjo; Zaal Kokaia; Oleg Shupliakov; Robert M Cassidy; Olle Lindvall; Jonas Frisén
Journal:  Nat Neurosci       Date:  2009-02-22       Impact factor: 24.884

3.  Canine ceroid-lipofuscinosis-a model for human neuronal ceroid-lipofuscinosis and aging.

Authors:  N Koppang
Journal:  Mech Ageing Dev       Date:  1973 Dec-1974 Feb       Impact factor: 5.432

4.  Enhanced survival of the LINCL mouse following CLN2 gene transfer using the rh.10 rhesus macaque-derived adeno-associated virus vector.

Authors:  Dolan Sondhi; Neil R Hackett; Daniel A Peterson; Jamie Stratton; Michael Baad; Kelly M Travis; James M Wilson; Ronald G Crystal
Journal:  Mol Ther       Date:  2006-12-19       Impact factor: 11.454

Review 5.  Genetics of the neuronal ceroid lipofuscinoses (Batten disease).

Authors:  Sara E Mole; Susan L Cotman
Journal:  Biochim Biophys Acta       Date:  2015-05-27

Review 6.  Neuronal ceroid-lipofuscinoses in childhood.

Authors:  P Santavuori
Journal:  Brain Dev       Date:  1988       Impact factor: 1.961

7.  AAV gene transfer delays disease onset in a TPP1-deficient canine model of the late infantile form of Batten disease.

Authors:  Martin L Katz; Luis Tecedor; Yonghong Chen; Baye G Williamson; Elena Lysenko; Fred A Wininger; Whitney M Young; Gayle C Johnson; Rebecca E H Whiting; Joan R Coates; Beverly L Davidson
Journal:  Sci Transl Med       Date:  2015-11-11       Impact factor: 17.956

8.  A frame shift mutation in canine TPP1 (the ortholog of human CLN2) in a juvenile Dachshund with neuronal ceroid lipofuscinosis.

Authors:  Tomoyuki Awano; Martin L Katz; Dennis P O'Brien; Istvan Sohar; Peter Lobel; Joan R Coates; Shahnawaz Khan; Gayle C Johnson; Urs Giger; Gary S Johnson
Journal:  Mol Genet Metab       Date:  2006-04-18       Impact factor: 4.204

9.  A reversal learning task detects cognitive deficits in a Dachshund model of late-infantile neuronal ceroid lipofuscinosis.

Authors:  D N Sanders; S Kanazono; F A Wininger; R E H Whiting; C A Flournoy; J R Coates; L J Castaner; D P O'Brien; M L Katz
Journal:  Genes Brain Behav       Date:  2011-08-07       Impact factor: 3.708

10.  Targeted gene transfer into ependymal cells through intraventricular injection of AAV1 vector and long-term enzyme replacement via the CSF.

Authors:  Yoshiyuki Yamazaki; Yukihiko Hirai; Koichi Miyake; Takashi Shimada
Journal:  Sci Rep       Date:  2014-07-01       Impact factor: 4.379
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