Literature DB >> 23622391

Neuronal ceroid lipofuscinoses.

Brigitte Chabrol1, Catherine Caillaud, Berge Minassian.   

Abstract

Neuronal ceroid lipofuscinoses (NCL) represent a group of autosomal recessive neurodegenerative disorders, presenting with myoclonic epilepsy, psychomotor delay, progressive loss of vision, and early death. Four main clinical forms have been delineated (infantile, late infantile, juvenile, and adult), but many other variants have also been described. At least 14 genetically distinct NCL, designated CLN1 to CLN14, are presently known.The identification of the deficient protein and/or the genetic defect is required for a specific diagnosis, which is necessary for a reliable genetic counseling in at-risk families.
Copyright © 2013 Elsevier B.V. All rights reserved.

Entities:  

Mesh:

Substances:

Year:  2013        PMID: 23622391     DOI: 10.1016/B978-0-444-59565-2.00038-1

Source DB:  PubMed          Journal:  Handb Clin Neurol        ISSN: 0072-9752


  11 in total

Review 1.  Molecular mechanisms of epilepsy.

Authors:  Kevin Staley
Journal:  Nat Neurosci       Date:  2015-02-24       Impact factor: 24.884

2.  Visual perception and macular integrity in non-classical CLN2 disease.

Authors:  Angela Schulz; Simon Dulz; Yevgeniya Atiskova; Jan Wildner; Eva Wibbeler; Miriam Nickel; Martin Stephan Spitzer; Christoph Schwering
Journal:  Graefes Arch Clin Exp Ophthalmol       Date:  2022-06-02       Impact factor: 3.535

Review 3.  Recent Insight into the Genetic Basis, Clinical Features, and Diagnostic Methods for Neuronal Ceroid Lipofuscinosis.

Authors:  Konrad Kaminiów; Sylwia Kozak; Justyna Paprocka
Journal:  Int J Mol Sci       Date:  2022-05-20       Impact factor: 6.208

4.  Neurophysiological and Genetic Findings in Patients With Juvenile Myoclonic Epilepsy.

Authors:  Stefani Stefani; Ioanna Kousiappa; Nicoletta Nicolaou; Eleftherios S Papathanasiou; Anastasis Oulas; Pavlos Fanis; Vassos Neocleous; Leonidas A Phylactou; George M Spyrou; Savvas S Papacostas
Journal:  Front Integr Neurosci       Date:  2020-08-20

Review 5.  Genetic forms of epilepsies and other paroxysmal disorders.

Authors:  Heather E Olson; Annapurna Poduri; Phillip L Pearl
Journal:  Semin Neurol       Date:  2014-09-05       Impact factor: 3.420

Review 6.  Moving towards effective therapeutic strategies for Neuronal Ceroid Lipofuscinosis.

Authors:  Ryan D Geraets; Seung yon Koh; Michelle L Hastings; Tammy Kielian; David A Pearce; Jill M Weimer
Journal:  Orphanet J Rare Dis       Date:  2016-04-16       Impact factor: 4.123

7.  Mice deficient in the lysosomal enzyme palmitoyl-protein thioesterase 1 (PPT1) display a complex retinal phenotype.

Authors:  Yevgeniya Atiskova; Susanne Bartsch; Tatyana Danyukova; Elke Becker; Christian Hagel; Stephan Storch; Udo Bartsch
Journal:  Sci Rep       Date:  2019-10-02       Impact factor: 4.379

8.  Detection of Infantile Batten Disease by Tandem Mass Spectrometry Assay of PPT1 Enzyme Activity in Dried Blood Spots.

Authors:  Hamid Khaledi; Yang Liu; Sophia Masi; Michael H Gelb
Journal:  Anal Chem       Date:  2018-09-24       Impact factor: 8.008

9.  A tailored mouse model of CLN2 disease: A nonsense mutant for testing personalized therapies.

Authors:  Ryan D Geraets; Logan M Langin; Jacob T Cain; Camille M Parker; Rosanna Beraldi; Attila D Kovacs; Jill M Weimer; David A Pearce
Journal:  PLoS One       Date:  2017-05-02       Impact factor: 3.752

10.  Searching for novel biomarkers using a mouse model of CLN3-Batten disease.

Authors:  Derek Timm; Jacob T Cain; Ryan D Geraets; Katherine A White; Seung Yon Koh; Tammy Kielian; David A Pearce; Michelle L Hastings; Jill M Weimer
Journal:  PLoS One       Date:  2018-08-07       Impact factor: 3.240

View more

北京卡尤迪生物科技股份有限公司 © 2022-2023.