Linear Diagnostic Procedure Elicited by Clinical Genetics and Validated by mRNA Analysis in Neuronal Ceroid Lipofuscinosis 7 Associated with a Novel Non-Canonical Splice Site Variant in


Journal

Genes
ISSN: 2073-4425
Titre abrégé: Genes (Basel)
Pays: Switzerland
ID NLM: 101551097

Informations de publication

Date de publication:
17 01 2023
Historique:
received: 09 12 2022
revised: 08 01 2023
accepted: 16 01 2023
entrez: 25 2 2023
pubmed: 26 2 2023
medline: 3 3 2023
Statut: epublish

Résumé

Neuronal ceroid lipofuscinoses (CNL) are lysosomal storage diseases that represent the most common cause of dementia in children. To date, 13 autosomal recessive (AR) and 1 autosomal dominant (AD) gene have been characterized. Biallelic variants in

Identifiants

pubmed: 36833170
pii: genes14020245
doi: 10.3390/genes14020245
pmc: PMC9956376
pii:
doi:

Substances chimiques

RNA, Messenger 0
DNA, Complementary 0
MFSD8 protein, human 0
Membrane Transport Proteins 0
SPATA5 protein, human 0
ATPases Associated with Diverse Cellular Activities EC 3.6.4.-

Types de publication

Case Reports Journal Article

Langues

eng

Sous-ensembles de citation

IM

Références

Neurogenetics. 2005 Sep;6(3):107-26
pubmed: 15965709
PLoS One. 2014 Oct 15;9(10):e109576
pubmed: 25333361
Biochim Biophys Acta. 2012 Oct;1822(10):1617-28
pubmed: 22668694
Int J Mol Sci. 2022 Feb 18;23(4):
pubmed: 35216386
Neurogenetics. 2009 Oct;10(4):307-11
pubmed: 19277732
Turk J Pediatr. 2004 Jan-Mar;46(1):1-10
pubmed: 15074367
Ophthalmic Genet. 2019 Apr;40(2):141-145
pubmed: 31006324
Eur J Paediatr Neurol. 2015 Jan;19(1):78-86
pubmed: 25439737
Brain. 2009 Mar;132(Pt 3):810-9
pubmed: 19201763
Neurol Sci. 2021 Mar;42(3):1103-1111
pubmed: 33486620
Front Neurol. 2022 Aug 02;13:907228
pubmed: 36034301
Neurogenetics. 2009 Feb;10(1):73-7
pubmed: 18850119
Pediatr Neurol. 2014 Jan;50(1):85-95
pubmed: 24120650
Eur J Med Genet. 2014 Nov-Dec;57(11-12):607-12
pubmed: 25270050
JIMD Rep. 2019 Sep 03;50(1):20-30
pubmed: 31741823
Clin Neurol Neurosurg. 2022 Sep;220:107375
pubmed: 35917699
Front Genet. 2022 Jan 26;13:807515
pubmed: 35154277
Ophthalmology. 2015 Jan;122(1):170-9
pubmed: 25227500
PLoS One. 2018 Dec 13;13(12):e0207958
pubmed: 30543658
Hum Mutat. 2012 Jan;33(1):42-63
pubmed: 21990111
N Engl J Med. 2019 Oct 24;381(17):1644-1652
pubmed: 31597037
Am J Hum Genet. 2007 Jul;81(1):136-46
pubmed: 17564970
Int J Mol Sci. 2022 Apr 13;23(8):
pubmed: 35457110
Biochim Biophys Acta. 2015 Oct;1852(10 Pt B):2256-61
pubmed: 26026924
Acta Neuropathol. 2019 Jan;137(1):71-88
pubmed: 30382371
Hum Mutat. 2009 Mar;30(3):E530-40
pubmed: 19177532
Genet Med. 2015 May;17(5):405-24
pubmed: 25741868
BMC Med Genet. 2018 Aug 25;19(1):151
pubmed: 30144815
Front Genet. 2019 Apr 25;10:370
pubmed: 31105743
Ophthalmic Genet. 2022 Jul 8;:1-5
pubmed: 35801630
Biochim Biophys Acta. 2013 Nov;1832(11):1795-800
pubmed: 22959893
Hum Mol Genet. 2010 Nov 15;19(22):4497-514
pubmed: 20826447
Biochim Biophys Acta. 2015 Oct;1852(10 Pt B):2262-6
pubmed: 25989315
J Med Case Rep. 2018 Sep 25;12(1):281
pubmed: 30249282
Nat Protoc. 2006;1(2):581-5
pubmed: 17406285
J Epilepsy Res. 2021 Jun 30;11(1):22-31
pubmed: 34395220
Invest Ophthalmol Vis Sci. 2017 Jun 1;58(7):2906-2914
pubmed: 28586915
Methods Mol Biol. 2012;867:49-63
pubmed: 22454054

Auteurs

Domizia Pasquetti (D)

Unit of Medical Genetics, Section of Genomic Medicine, Department of Life Sciences and Public Health, Università Cattolica del Sacro Cuore, Fondazione Policlinico Universitario A. Gemelli IRCCS, 00168 Rome, Italy.

Giuseppe Marangi (G)

Unit of Medical Genetics, Section of Genomic Medicine, Department of Life Sciences and Public Health, Università Cattolica del Sacro Cuore, Fondazione Policlinico Universitario A. Gemelli IRCCS, 00168 Rome, Italy.

Daniela Orteschi (D)

Unit of Medical Genetics, Section of Genomic Medicine, Department of Life Sciences and Public Health, Università Cattolica del Sacro Cuore, Fondazione Policlinico Universitario A. Gemelli IRCCS, 00168 Rome, Italy.

Marina Carapelle (M)

Unit of Medical Genetics, Section of Genomic Medicine, Department of Life Sciences and Public Health, Università Cattolica del Sacro Cuore, Fondazione Policlinico Universitario A. Gemelli IRCCS, 00168 Rome, Italy.

Federica Francesca L'Erario (FF)

Unit of Medical Genetics, Section of Genomic Medicine, Department of Life Sciences and Public Health, Università Cattolica del Sacro Cuore, Fondazione Policlinico Universitario A. Gemelli IRCCS, 00168 Rome, Italy.

Romina Venditti (R)

Child Neurology and Psychiatry Unit, Department of Neuroscience, Catholic University of the Sacred Heart, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, 00168 Rome, Italy.

Sabrina Maietta (S)

Unit of Medical Genetics, Section of Genomic Medicine, Department of Life Sciences and Public Health, Università Cattolica del Sacro Cuore, Fondazione Policlinico Universitario A. Gemelli IRCCS, 00168 Rome, Italy.

Domenica Immacolata Battaglia (DI)

Child Neurology and Psychiatry Unit, Department of Neuroscience, Catholic University of the Sacred Heart, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, 00168 Rome, Italy.

Ilaria Contaldo (I)

Child Neurology and Psychiatry Unit, Department of Neuroscience, Catholic University of the Sacred Heart, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, 00168 Rome, Italy.

Chiara Veredice (C)

Child Neurology and Psychiatry Unit, Department of Neuroscience, Catholic University of the Sacred Heart, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, 00168 Rome, Italy.

Marcella Zollino (M)

Unit of Medical Genetics, Section of Genomic Medicine, Department of Life Sciences and Public Health, Università Cattolica del Sacro Cuore, Fondazione Policlinico Universitario A. Gemelli IRCCS, 00168 Rome, Italy.

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Classifications MeSH