Genetic and Clinical Heterogeneity in Thirteen New Cases with Aceruloplasminemia. Atypical Anemia as a Clue for an Early Diagnosis.


Journal

International journal of molecular sciences
ISSN: 1422-0067
Titre abrégé: Int J Mol Sci
Pays: Switzerland
ID NLM: 101092791

Informations de publication

Date de publication:
30 Mar 2020
Historique:
received: 12 03 2020
accepted: 26 03 2020
entrez: 3 4 2020
pubmed: 3 4 2020
medline: 5 1 2021
Statut: epublish

Résumé

Aceruloplasminemia is a rare autosomal recessive genetic disease characterized by mild microcytic anemia, diabetes, retinopathy, liver disease, and progressive neurological symptoms due to iron accumulation in pancreas, retina, liver, and brain. The disease is caused by mutations in the Ceruloplasmin (

Identifiants

pubmed: 32235485
pii: ijms21072374
doi: 10.3390/ijms21072374
pmc: PMC7178074
pii:
doi:

Substances chimiques

Ceruloplasmin EC 1.16.3.1

Types de publication

Journal Article

Langues

eng

Sous-ensembles de citation

IM

Subventions

Organisme : Veneto Region
ID : PRIHTA no.2014-00000451
Organisme : Spanish Secretary of Research, Development and Innovation (MINECO) Spain
ID : SAF2015-70412-R
Organisme : Fundação de Apoio à Pesquisa do Estado de São Paulo (FAPESP)
ID : 2016/08072-9
Organisme : Fondazione Cariverona
ID : 2014.0851
Organisme : Wellcome Trust
Pays : United Kingdom
Organisme : Fundação de Apoio à Pesquisa do Estado de São Paulo (FAPESP)
ID : 2014/00984-3

Références

J Neurol Neurosurg Psychiatry. 2008 Apr;79(4):467-70
pubmed: 17911185
Annu Rev Nutr. 2002;22:439-58
pubmed: 12055353
Biometals. 2003 Mar;16(1):205-13
pubmed: 12572680
Mol Genet Genomic Med. 2015 May;3(3):221-32
pubmed: 26029709
J Comput Biol. 1997 Fall;4(3):311-23
pubmed: 9278062
Bioinformatics. 2009 May 1;25(9):1189-91
pubmed: 19151095
FASEB J. 2019 Dec;33(12):13492-13502
pubmed: 31560858
Nucleic Acids Res. 2004 Mar 19;32(5):1792-7
pubmed: 15034147
Nucleic Acids Res. 2016 Jul 8;44(W1):W344-50
pubmed: 27166375
Am J Hematol. 2017 Jun;92(6):562-568
pubmed: 28335084
Neurology. 1987 May;37(5):761-7
pubmed: 3574673
Acta Crystallogr D Biol Crystallogr. 2007 Feb;63(Pt 2):240-8
pubmed: 17242517
J Comput Chem. 2004 Oct;25(13):1605-12
pubmed: 15264254
Nucleic Acids Res. 2010 Jan;38(Database issue):D211-22
pubmed: 19920124
Front Neurosci. 2019 Apr 05;13:325
pubmed: 31024241
Am J Hematol. 2016 Jun;91(4):420-5
pubmed: 26799139
Blood. 2018 Feb 22;131(8):899-910
pubmed: 29237594
Nucleic Acids Res. 1992 Nov 11;20(21):5533-40
pubmed: 1454517
Cell. 2017 Jan 26;168(3):344-361
pubmed: 28129536
Blood. 2014 Jun 19;123(25):3873-86; quiz 4005
pubmed: 24665134
Ann Neurol. 1997 Mar;41(3):404-7
pubmed: 9066364
Biochim Biophys Acta. 2010 Nov;1802(11):968-75
pubmed: 20655381
Neuropathology. 2015 Feb;35(1):83-90
pubmed: 25168455
Cell Metab. 2015 Nov 3;22(5):777-87
pubmed: 26437604
Eur Neurol. 1999;42(3):157-62
pubmed: 10529542
Genet Med. 2015 May;17(5):405-24
pubmed: 25741868
Br J Haematol. 2013 Jan;160(1):12-24
pubmed: 23057559
J Biol Chem. 2000 Feb 11;275(6):4305-10
pubmed: 10660599
Curr Neuropharmacol. 2019;17(6):539-549
pubmed: 29737252
PLoS One. 2013 Jul 03;8(7):e67145
pubmed: 23843990
Mutat Res. 2018 Jul - Sep;777:52-63
pubmed: 30115430
Int Rev Neurobiol. 2013;110:125-51
pubmed: 24209437
Nat Methods. 2010 Apr;7(4):248-9
pubmed: 20354512
EMBO Mol Med. 2018 Jan;10(1):91-106
pubmed: 29183916
Parkinsonism Relat Disord. 2017 Mar;36:33-40
pubmed: 28012953
Nucleic Acids Res. 2009 May;37(9):e67
pubmed: 19339519
Science. 2004 Dec 17;306(5704):2090-3
pubmed: 15514116
Physiol Rev. 2013 Oct;93(4):1721-41
pubmed: 24137020
Front Neurosci. 2018 Dec 04;12:903
pubmed: 30568573
Brain Dev. 2007 Aug;29(7):450-3
pubmed: 17307325
Parkinsonism Relat Disord. 2018 Jun;51:36-42
pubmed: 29503155
Nat Protoc. 2009;4(7):1073-81
pubmed: 19561590
J Biol Chem. 1991 Mar 15;266(8):5128-34
pubmed: 2002050

Auteurs

Marc Vila Cuenca (M)

Iron Metabolism: Regulation and Diseases Group, Josep Carreras Leukaemia Research Institute (IJC), Campus Can Ruti, Badalona, 08916 Barcelona, Spain.

Giacomo Marchi (G)

EuroBloodNet Referral Center for Iron Disorders and Gruppo Interdisciplinare Malattie del Ferro, Internal Medicine Unit, Azienda Ospedaliera Universitaria Integrata di Verona, 37134 Verona, Italy.

Anna Barqué (A)

Iron Metabolism: Regulation and Diseases Group, Josep Carreras Leukaemia Research Institute (IJC), Campus Can Ruti, Badalona, 08916 Barcelona, Spain.

Clara Esteban-Jurado (C)

Iron Metabolism: Regulation and Diseases Group, Josep Carreras Leukaemia Research Institute (IJC), Campus Can Ruti, Badalona, 08916 Barcelona, Spain.

Alessandro Marchetto (A)

Department of Biotechnology, University of Verona, 37134 Verona, Italy.

Alejandro Giorgetti (A)

Department of Biotechnology, University of Verona, 37134 Verona, Italy.

Viorica Chelban (V)

National Hospital for Neurology and Neurosurgery, London WC1N 3BG, UK.
Department of Neuromuscular Diseases, Institute of Neurology, University College London, London WC1N 3BG, UK.
Department of Neurology and Neurosurgery, Institute of Emergency Medicine, Toma Ciorbă 1, Chisinau, MD-2052 Chisinau, Republic of Moldova.

Henry Houlden (H)

National Hospital for Neurology and Neurosurgery, London WC1N 3BG, UK.
Department of Neuromuscular Diseases, Institute of Neurology, University College London, London WC1N 3BG, UK.
Neurogenetics Laboratory, The National Hospital for Neurology and Neurosurgery, London WC1N 3BG, UK.

Nicholas W Wood (NW)

National Hospital for Neurology and Neurosurgery, London WC1N 3BG, UK.
Neurogenetics Laboratory, The National Hospital for Neurology and Neurosurgery, London WC1N 3BG, UK.

Chiara Piubelli (C)

Centre for Tropical Diseases, Ospedale Sacro Cuore - Don Calabria, 37024 Negrar (VR), Italy.

Marina Dorigatti Borges (M)

Hematology and Hemotherapy Center-Hemocentro Campinas, University of Campinas-UNICAMP, Campinas 13083-878, Brazil.

Dulcinéia Martins de Albuquerque (D)

Hematology and Hemotherapy Center-Hemocentro Campinas, University of Campinas-UNICAMP, Campinas 13083-878, Brazil.

Kleber Yotsumoto Fertrin (K)

Hematology and Hemotherapy Center-Hemocentro Campinas, University of Campinas-UNICAMP, Campinas 13083-878, Brazil.
Division of Hematology, Department of Medicine, University of Washington, Seattle, WA 98195, USA.

Ester Jové-Buxeda (E)

Internal Medicine Department, Parc Tauli Hospital Universitari, Institut d' Investigació i Innovació Parc Tauli I3PT, Universidad Autonoma de Barcelona, 08208 Sabadell, Spain.

Jordi Sanchez-Delgado (J)

Hepatology Unit, Digestive Diseases Department, Parc Tauli Hospital Universitari. Institut d' Investigació i Innovació Parc Tauli I3PT, Universidad Autonoma de Barcelona, 08208 Sabadell, Spain.
Centro de Investigación Biomedica y en red Enfermedades hepáticas y digestivas (CIBERehd), Instituto de Salud Carlos III, 28029 Madrid, Spain.

Neus Baena-Díez (N)

Genetic Department, Parc Tauli Hospital Universitari, Institut d' Investigació i Innovació Parc Tauli I3PT, Universidad Autonoma de Barcelona, 08208 Sabadell, Spain.

Birute Burnyte (B)

Department of Human and Medical Genetics, Institute of Biomedical Sciences, Faculty of Medicine, Vilnius University, LT-08661 Vilnius, Lithuania.

Algirdas Utkus (A)

Department of Human and Medical Genetics, Institute of Biomedical Sciences, Faculty of Medicine, Vilnius University, LT-08661 Vilnius, Lithuania.

Fabiana Busti (F)

EuroBloodNet Referral Center for Iron Disorders and Gruppo Interdisciplinare Malattie del Ferro, Internal Medicine Unit, Azienda Ospedaliera Universitaria Integrata di Verona, 37134 Verona, Italy.

Gintaras Kaubrys (G)

Clinic of Neurology and Neurosurgery, Institute of Clinical Medicine, Faculty of Medicine, Vilnius University, 08661 Vilnius, Lithuania.

Eda Suku (E)

Department of Biotechnology, University of Verona, 37134 Verona, Italy.

Kamil Kowalczyk (K)

Department of Adult Neurology, Medical University of Gdańsk, 80-210 Gdańsk, Poland.

Bartosz Karaszewski (B)

Department of Adult Neurology, Medical University of Gdańsk, 80-210 Gdańsk, Poland.

John B Porter (JB)

Joint Red Cell Unit, Haematology Department, University College London NHS Foundation Trust, Cancer Services, 250 Euston Road, London NW1 2PG, UK.

Sally Pollard (S)

Consultant Paediatrician, Bradford Royal Infirmary, Duckworthlane, Bradford BD9 6RJ, UK.

Perla Eleftheriou (P)

Joint Red Cell Unit, Haematology Department, University College London NHS Foundation Trust, Cancer Services, 250 Euston Road, London NW1 2PG, UK.

Patricia Bignell (P)

Oxford Regional Genetics Laboratory, Oxford University Hospitals NHS Foundation Trust, The Churchill Hospital, Oxford OX3 7LE, UK.

Domenico Girelli (D)

EuroBloodNet Referral Center for Iron Disorders and Gruppo Interdisciplinare Malattie del Ferro, Internal Medicine Unit, Azienda Ospedaliera Universitaria Integrata di Verona, 37134 Verona, Italy.

Mayka Sanchez (M)

Iron Metabolism: Regulation and Diseases Group, Department of Basic Sciences, Faculty of Medicine and Health Sciences, Universitat Internacional de Catalunya (UIC); Sant Cugat del Valles, 08017 Barcelona, Spain.
Program of Program of Predictive and Personalized Medicine of Cancer (PMPPC), Institut d 'Investigació Germans Trias i Pujol (IGTP), Campus Can Ruti, Badalona, 08916 Barcelona, Spain.
BloodGenetics S.L., Esplugues de Llobregat, 08950 Barcelona, Spain.

Articles similaires

[Redispensing of expensive oral anticancer medicines: a practical application].

Lisanne N van Merendonk, Kübra Akgöl, Bastiaan Nuijen
1.00
Humans Antineoplastic Agents Administration, Oral Drug Costs Counterfeit Drugs

Smoking Cessation and Incident Cardiovascular Disease.

Jun Hwan Cho, Seung Yong Shin, Hoseob Kim et al.
1.00
Humans Male Smoking Cessation Cardiovascular Diseases Female
Humans United States Aged Cross-Sectional Studies Medicare Part C
1.00
Humans Yoga Low Back Pain Female Male

Classifications MeSH