TY - JOUR
T1 - De novo GRIN1 mutations
T2 - An emerging cause of severe early infantile encephalopathy
AU - Zehavi, Yoav
AU - Mandel, Hanna
AU - Zehavi, Arie
AU - Rashid, Muhammad Abu
AU - Straussberg, Rachel
AU - Jabur, Banan
AU - Shaag, Avraham
AU - Elpeleg, Orly
AU - Spiegel, Ronen
N1 - Publisher Copyright:
© 2017 Elsevier Masson SAS
PY - 2017/6/1
Y1 - 2017/6/1
N2 - De novo GRIN1 mutations have recently been shown to cause severe intellectual disability, hypotonia, hyperkinetic and stereotyped movements, and epilepsy. We report two new cases of severe early onset encephalopathy associated with hyperkinetic and oculogyric-like movements, caused by mutations in the GRIN1 gene; both were identified by whole exome sequencing. One of the patients harbored the novel mutation p.Ser688Tyr and the other patient harbored the p.Gly827Arg mutation, which was previously reported in three patients. In silico studies suggested that the p.Se688Tyr mutation results in disruption of NMDA ligand binding and the p.Gly827Arg mutation results in disrupted gating of the ion channel. Our study highlights the importance of GRIN1 mutations in the etiology of isolated cases of early onset encephalopathy, and the valuable role of whole exome sequencing in identifying these mutations.
AB - De novo GRIN1 mutations have recently been shown to cause severe intellectual disability, hypotonia, hyperkinetic and stereotyped movements, and epilepsy. We report two new cases of severe early onset encephalopathy associated with hyperkinetic and oculogyric-like movements, caused by mutations in the GRIN1 gene; both were identified by whole exome sequencing. One of the patients harbored the novel mutation p.Ser688Tyr and the other patient harbored the p.Gly827Arg mutation, which was previously reported in three patients. In silico studies suggested that the p.Se688Tyr mutation results in disruption of NMDA ligand binding and the p.Gly827Arg mutation results in disrupted gating of the ion channel. Our study highlights the importance of GRIN1 mutations in the etiology of isolated cases of early onset encephalopathy, and the valuable role of whole exome sequencing in identifying these mutations.
KW - De novo mutation
KW - Early onset encephalopathy
KW - GRIN1
KW - Oculogyric movements
UR - http://www.scopus.com/inward/record.url?scp=85017144392&partnerID=8YFLogxK
U2 - 10.1016/j.ejmg.2017.04.001
DO - 10.1016/j.ejmg.2017.04.001
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C2 - 28389307
AN - SCOPUS:85017144392
SN - 1769-7212
VL - 60
SP - 317
EP - 320
JO - European Journal of Medical Genetics
JF - European Journal of Medical Genetics
IS - 6
ER -