Not only dominant, not only optic atrophy: expanding the clinical spectrum associated with OPA1 mutations

被引:0
作者
Alessia Nasca
Teresa Rizza
Mara Doimo
Andrea Legati
Andrea Ciolfi
Daria Diodato
Cristina Calderan
Gianfranco Carrara
Eleonora Lamantea
Chiara Aiello
Michela Di Nottia
Marcello Niceta
Costanza Lamperti
Anna Ardissone
Stefania Bianchi-Marzoli
Giancarlo Iarossi
Enrico Bertini
Isabella Moroni
Marco Tartaglia
Leonardo Salviati
Rosalba Carrozzo
Daniele Ghezzi
机构
[1] Fondazione IRCCS Istituto Neurologico ‘Carlo Besta’,Unit of Molecular Neurogenetics
[2] Bambino Gesù Children’s Hospital,Unit of Muscular and Neurodegenerative Disorders, Laboratory of Molecular Medicine
[3] IRCCS,Department of Woman and Child Health, Clinical Genetics Unit
[4] University of Padova,Istituto di Ricerca Pediatria, IRP
[5] Città della Speranza,Division of Genetics and Rare Diseases, Molecular Genetics and Genomics Unit
[6] Bambino Gesù Children’s Hospital,Unit of Child Neurology
[7] IRCCS,Department of Ophthalmology, Neuro
[8] Fondazione IRCCS Istituto Neurologico ‘Carlo Besta’,ophthalmology Unit
[9] IRCCS Istituto Auxologico Italiano,Department of Ophthalmology
[10] Bambino Gesù IRCSS Children’s Hospital,undefined
来源
Orphanet Journal of Rare Diseases | / 12卷
关键词
OPA1; Optic atrophy; Mitochondrial disorder; Encephalopathy; Recessive trait; Targeted resequencing; WES;
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[1]  
Amati-Bonneau P(2005)OPA1 R445H mutation in optic atrophy associated with sensorineural deafness Ann Neurol 58 958-963
[2]  
Guichet A(2008)OPA1 mutations induce mitochondrial DNA instability and optic atrophy ‘plus’ phenotypes Brain 131 338-351
[3]  
Olichon A(2010)Multi-system neurological disease is common in patients with OPA1 mutations Brain 133 771-786
[4]  
Chevrollier A(2001)OPA1 mutations in patients with autosomal dominant optic atrophy and evidence for semi-dominant inheritance Hum Molec Genet 10 1359-1368
[5]  
Viala F(2011)Early-onset severe neuromuscular phenotype associated with compound heterozygosity for OPA1 mutations Molec Genet Metab 103 383-387
[6]  
Miot S(2014)Early-onset Behr syndrome due to compound heterozygous mutations in OPA1 Brain 137 e301-131
[7]  
Ayuso C(2015)‘Behr syndrome’ with OPA1 compound heterozygote mutations Brain 138 e321-95
[8]  
Odent S(2016)Fatal infantile mitochondrial encephalomyopathy, hypertrophic cardiomyopathy and optic atrophy associated with a homozygous OPA1 mutation J Med Genet 53 127-35
[9]  
Arrouet C(2002)Detection and quantification of mitochondrial DNA deletions in individual cells by real-time PCR Nucleic Acids Res 30 e68-303
[10]  
Amati-Bonneau P(2013)Mutations in FBXL4, encoding a mitochondrial protein, cause early-onset mitochondrial encephalomyopathy Am J Hum Genet 93 482-7