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Human Mutation
Article . 2014
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Mutation in theSYNJ1Gene Associated with Autosomal Recessive, Early-Onset Parkinsonism

Authors: Quadri M; Fang M; Picillo M; Olgiati S; Breedveld GJ; Graafland J; Wu B; +91 Authors

Mutation in theSYNJ1Gene Associated with Autosomal Recessive, Early-Onset Parkinsonism

Abstract

Autosomal recessive, early-onset Parkinsonism is clinically and genetically heterogeneous. Here, we report the identification, by homozygosity mapping and exome sequencing, of a SYNJ1 homozygous mutation (p.Arg258Gln) segregating with disease in an Italian consanguineous family with Parkinsonism, dystonia, and cognitive deterioration. Response to levodopa was poor, and limited by side effects. Neuroimaging revealed brain atrophy, nigrostriatal dopaminergic defects, and cerebral hypometabolism. SYNJ1 encodes synaptojanin 1, a phosphoinositide phosphatase protein with essential roles in the postendocytic recycling of synaptic vesicles. The mutation is absent in variation databases and in ethnically matched controls, is damaging according to all prediction programs, and replaces an amino acid that is extremely conserved in the synaptojanin 1 homologues and in SAC1-like domains of other proteins. Sequencing the SYNJ1 ORF in unrelated patients revealed another heterozygous mutation (p.Ser1422Arg), predicted as damaging, in a patient who also carries a heterozygous PINK1 truncating mutation. The SYNJ1 gene is a compelling candidate for Parkinsonism; mutations in the functionally linked protein auxilin cause a similar early-onset phenotype, and other findings implicate endosomal dysfunctions in the pathogenesis. Our data delineate a novel form of human Mendelian Parkinsonism, and provide further evidence for abnormal synaptic vesicle recycling as a central theme in the pathogenesis.

Keywords

Adult, Male, Adolescent, Auxilins, Genes, Recessive, DCN PAC - Perception action and control, Parkinsonism, DCN MP - Plasticity and memory NCEBP 10: Human Movement & Fatigue, SYNJ1, Levodopa, Consanguinity, SDG 3 - Good Health and Well-being, Parkinsonian Disorders, Humans, Exome, Age of Onset, gene, Child, Parkinson’s disease; Early onset; EOPD; SYNJ1Gene, EMC MGC-02-96-01, PINK1, Homozygote, Genetic Variation, Middle Aged, Dystonia, Italy, Mutation, Dementia; Dystonia; Gene; Mutation; Parkinsonism; PINK1; SYNJ1, Female, dystonia, PINK1; Parkinsonism; SYNJ1; dementia; dystonia; gene; mutation, mutation, Cognition Disorders, dementia

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citations
This is an alternative to the "Influence" indicator, which also reflects the overall/total impact of an article in the research community at large, based on the underlying citation network (diachronically).
BIP!Citations provided by BIP!
popularity
This indicator reflects the "current" impact/attention (the "hype") of an article in the research community at large, based on the underlying citation network.
BIP!Popularity provided by BIP!
influence
This indicator reflects the overall/total impact of an article in the research community at large, based on the underlying citation network (diachronically).
BIP!Influence provided by BIP!
impulse
This indicator reflects the initial momentum of an article directly after its publication, based on the underlying citation network.
BIP!Impulse provided by BIP!
285
Top 1%
Top 1%
Top 1%