Aicardi-Goutieres syndrome and related phenotypes: linking nucleic acid metabolism with autoimmunityYanick J Crow
Academic Unit of Medical Genetics, Manchester Academic Health Science Centre, Central Manchester Foundation Trust, St Mary s Hospital, University of Manchester, Oxford Road, Manchester M13 9WL, UK
Hum Mol Genet 18:R130-6. 2009
..These studies provide important insights into the pathogenesis of SLE and beg urgent questions about the development and use of immunosuppressive therapies in AGS and related phenotypes...
Aicardi-Goutières syndromeYanick J Crow
Genetic Medicine, University of Manchester, St Mary s Hospital, Manchester, UK Electronic address
Handb Clin Neurol 113:1629-35. 2013
..These studies beg urgent questions about the development and use of immunosuppressive therapies in AGS and related phenotypes...
Type I interferonopathies: a novel set of inborn errors of immunityYanick J Crow
Genetic Medicine, University of Manchester, Manchester Academic Health Science Centre, Central Manchester Foundation Trust University Hospitals, United Kingdom
Ann N Y Acad Sci 1238:91-8. 2011
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The story of DNase II: a stifled death-wish leads to self-harmYanick J Crow
Genetic Medicine, University of Manchester, Manchester Academic Health Science Centre, Central Manchester Foundation Trust University Hospitals, Manchester, UK
Eur J Immunol 40:2376-8. 2010
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Aicardi-Goutières syndrome: an important Mendelian mimic of congenital infectionYanick J Crow
Wellcome Trust Brenner Building, Leeds Institute of Molecular Medicine, St James s University Hospital, Leeds, West Yorkshire, UK
Dev Med Child Neurol 50:410-6. 2008
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Mutations in the gene encoding the 3'-5' DNA exonuclease TREX1 cause Aicardi-Goutières syndrome at the AGS1 locusYanick J Crow
Leeds Institute of Molecular Medicine, University of Leeds, St James s University Hospital, Leeds LS9 7TF, UK
Nat Genet 38:917-20. 2006
..Our findings suggest an unanticipated role for TREX1 in processing or clearing anomalous DNA structures, failure of which results in the triggering of an abnormal innate immune response...
Mutations in genes encoding ribonuclease H2 subunits cause Aicardi-Goutières syndrome and mimic congenital viral brain infectionYanick J Crow
Leeds Institute of Molecular Medicine, University of Leeds, St James s University Hospital, Leeds, LS9 7TF, UK
Nat Genet 38:910-6. 2006
..Our findings demonstrate a role for ribonuclease H in human neurological disease and suggest an unanticipated relationship between ribonuclease H2 and the antiviral immune response that warrants further investigation...
Clinical and molecular phenotype of Aicardi-Goutieres syndromeGillian Rice
Leeds Institute of Molecular Medicine, St James s University Hospital, Leeds, LS9 7TF, UK
Am J Hum Genet 81:713-25. 2007
..Our analysis defines the phenotypic spectrum of AGS and suggests a coherent mutation-screening strategy in this heterogeneous disorder. Additionally, our data indicate that at least one further AGS-causing gene remains to be identified...
Mutations in ADAR1 cause Aicardi-Goutières syndrome associated with a type I interferon signatureGillian I Rice
Manchester Academic Health Science Centre, University of Manchester, Genetic Medicine, UK
Nat Genet 44:1243-8. 2012
..Considering recent insights derived from the study of other AGS-related proteins, we speculate that ADAR1 may limit the cytoplasmic accumulation of the dsRNA generated from genomic repetitive elements...
Newly recognized recessive syndrome characterized by dysmorphic features, hypogonadotropic hypogonadism, severe microcephaly, and sensorineural hearing loss maps to 3p21.3Emma M Jenkinson
Genetic Medicine, Manchester Academic Health Sciences Centre MAHSC, University of Manchester and Central Manchester University Hospitals NHS Foundation Trust, Manchester, UK
Am J Med Genet A 155:2910-5. 2011
..1 Mb on chromosome 3p21 common to the affected individuals. The critical region contains 227 genes and initial sequence analysis of a functional candidate gene has not identified causative mutations...
Recognizable phenotypes associated with intracranial calcificationJohn H Livingston
Department of Paediatric Neurology, Leeds General Infirmary, Leeds, UK
Dev Med Child Neurol 55:46-57. 2013
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Heterozygous mutations in TREX1 cause familial chilblain lupus and dominant Aicardi-Goutieres syndromeGillian Rice
Leeds Institute of Molecular Medicine, St James s University Hospital, Leeds, LS9 7TF, UK
Am J Hum Genet 80:811-5. 2007
..Additionally, we describe a de novo heterozygous mutation, affecting a critical catalytic residue in TREX1, that results in typical Aicardi-Goutieres syndrome...
Focal dermal hypoplasia with subependymal heterotopia and hypoplastic corpus callosumRichard B Fisher
Department of Clinical Genetics, St James s University Hospital, Leeds, UK
Clin Dysmorphol 16:59-61. 2007
..However, none of these conditions, fully explains the collection of abnormalities found in this patient...
How genetically heterogeneous is Kabuki syndrome?: MLL2 testing in 116 patients, review and analyses of mutation and phenotypic spectrumSiddharth Banka
Department of Genetic Medicine, St Mary s Hospital, Manchester Academic Health Sciences Centre MAHSC, University of Manchester, Manchester, UK
Eur J Hum Genet 20:381-8. 2012
..Moreover, phenotypic variability of KS suggests that MLL2 testing should be considered even in atypical patients...
Severe neonatal-onset panniculitis in a female infant with Prader-Willi syndromeMuthukumar Sakthivel
Department of Genetic Medicine, Central Manchester Foundation Trust University Hospitals, Manchester Academic Health Science Centre, University of Manchester, Manchester, UK
Am J Med Genet A 155:3087-9. 2011
..Such an association might be explained by the unmasking of a recessive allele as a consequence of hemizygosity, in the case of a 15q11 deletion, or homozygosity, in the case of maternal isodisomy...
Mutations in CTC1, encoding conserved telomere maintenance component 1, cause Coats plusBeverley H Anderson
Manchester Academic Health Science Centre, University of Manchester, Genetic Medicine, UK
Nat Genet 44:338-42. 2012
..Thus, CTC1 may have a function in DNA metabolism that is necessary for but not specific to telomeric integrity...
Brown-Vialetto-Van Laere syndrome; variability in age at onset and disease progression highlighting the phenotypic overlap with Fazio-Londe diseaseSubrahmanian Dipti
Department of Paediatric Neurology, Leeds General Infirmary, Leeds, UK
Brain Dev 27:443-6. 2005
..Rather than representing two separate disorders, our findings suggest the possibility of a single disease entity which may usefully be considered a form of juvenile amyotrophic lateral sclerosis...
Further delineation of the phenotype of severe congenital neutropenia type 4 due to mutations in G6PC3Siddharth Banka
Genetic Medicine, Manchester Academic Health Sciences Centre MAHSC, University of Manchester, St Mary s Hospital, Manchester, UK
Eur J Hum Genet 19:18-22. 2011
..Delineation of the SCN4 phenotype may help in appropriate treatment and management and provide further insights into the pathogenesis of this multisystem disease...
Recessive mutations in the gene encoding the tight junction protein occludin cause band-like calcification with simplified gyration and polymicrogyriaMary C O'Driscoll
University of Manchester, Manchester Academic Health Science Centre, Central Manchester Foundation Trust University Hospitals, UK
Am J Hum Genet 87:354-64. 2010
..We provide evidence that the tight junction protein occludin (encoded by the OCLN gene) is involved in the pathogenesis of malformations of cortical development...
Identification and characterization of an inborn error of metabolism caused by dihydrofolate reductase deficiencySiddharth Banka
Genetic Medicine, Manchester Academic Health Sciences Centre MAHSC, St Mary s Hospital, University of Manchester, Manchester, UK
Am J Hum Genet 88:216-25. 2011
..Moreover, this relationship provides insight into the role of folates in neurological conditions, including depression, Alzheimer disease, and Parkinson disease...
Intracranial calcification in early infantile Krabbe disease: nothing new under the sunJohn H Livingston
Department of Paediatric Neurology, Leeds General Infirmary, Leeds, UK
Dev Med Child Neurol 54:376-9. 2012
..This report serves as a reminder that Krabbe disease should be included in the differential diagnosis of disorders causing intracranial calcification...
Infantile neurological Degos diseaseTong Hong Yeo
Department of Paediatric Neurology, Royal Manchester Children s Hospital, Manchester M13 9WL, UK
Eur J Paediatr Neurol 15:167-70. 2011
..CT scan revealed intracranial calcification, a feature described in two previous cases. Our report highlights the need to consider Degos disease in the differential diagnosis of childhood neurological disease with skin involvement...
Tartrate-resistant acid phosphatase deficiency causes a bone dysplasia with autoimmunity and a type I interferon expression signatureTracy A Briggs
Manchester Academic Heath Science Centre, University of Manchester, Genetic Medicine, Manchester, UK
Nat Genet 43:127-31. 2011
..Our findings reveal a previously unrecognized link between tartrate-resistant acid phosphatase activity and interferon metabolism and highlight the importance of type I interferon in the genesis of autoimmunity...
Diagnosing fetal alcohol syndrome: new insights from newer genetic technologiesSofia Douzgou
Clinical Fellow in Clinical Genetics, Central Manchester University Hospitals Foundation Trust, Manchester Academic Health Sciences Centre, Genetic Medicine, St Mary s Hospital, Oxford Rd, Manchester, UK
Arch Dis Child 97:812-7. 2012
..We studied the outcome of genetic assessment of 80 children referred to a regional genetics centre between 2004 and 2010 to identify the value of the genetic assessment in cases of suspected FASD...
Congenital glaucoma and brain stem atrophy as features of Aicardi-Goutières syndromeYanick J Crow
Molecular Medicine Unit, University of Leeds, St James s University Hospital, Leeds, United Kingdom
Am J Med Genet A 129:303-7. 2004
..Additionally, neuroimaging demonstrated significant brain stem atrophy in the affected sib-pair. These features have not been previously described in Aicardi-Goutières syndrome and expand the phenotypic spectrum...
Sedaghatian spondylometaphyseal dysplasia with pachygyria and absence of the corpus callosumSharon J English
Department of Neonatal Medicine, Leeds General Infirmary, Leeds, United Kingdom
Am J Med Genet A 140:1854-8. 2006
..Our case highlights the possibility for neuronal migration abnormalities in Sedaghatian spondylometaphyseal dysplasia and suggests a novel association of the disorder with agenesis of the corpus callosum...
Aicardi-Goutières syndrome: description of a late onset caseStefano D'Arrigo
Department of Developmental Neurology, Fondazione IRCCS Istituto Neurologico C Besta, Milan, Italy
Dev Med Child Neurol 50:631-4. 2008
..Our paper also considers the possible value of immunomodulatory therapy in AGS...
Clinical and mutational spectrum of Mowat-Wilson syndromeChristiane Zweier
Institute of Human Genetics, Friedrich Alexander University Erlangen Nuremberg, Erlangen, Germany
Eur J Med Genet 48:97-111. 2005
..The lack of missense mutations in MWS and MWS-like patients suggests there may be other, as yet unrecognized phenotypes, associated with missense mutations of this transcription factor...
Congenital palmar polyonychia with postaxial limb defects may be the same as the ulnar-mammary syndromeYanick J Crow
Am J Med Genet A 137:233; author reply 234. 2005
Mutations in the pericentrin (PCNT) gene cause primordial dwarfismAnita Rauch
Institute of Human Genetics, University Hospital Erlangen, Friedrich Alexander University Erlangen Nuremberg, Erlangen, Germany
Science 319:816-9. 2008
..Absence of PCNT results in disorganized mitotic spindles and missegregation of chromosomes. Mutations in related genes are known to cause primary microcephaly (MCPH1, CDK5RAP2, ASPM, and CENPJ)...
Identification of microcephalin, a protein implicated in determining the size of the human brainAndrew P Jackson
Molecular Medicine Unit, University of Leeds, United Kingdom
Am J Hum Genet 71:136-42. 2002
..This gene, microcephalin, is expressed in the developing cerebral cortex of the fetal brain. Further study of this and related genes may provide important new insights into neocortical development and evolution...