A nonsense mutation of PEPD in four Amish children with prolidase deficiencyHeng Wang
Das Deutsch Center DDC Clinic for Special Needs Children, Middlefield, Ohio 44062, USA
Am J Med Genet A 140:580-5. 2006
..793 T > C in exon 11, resulting in a premature stop-codon at amino acid residue 265 (p.R265X). It is speculated that the severe phenotype in these patients might be associated with the type of the PEPD gene mutation...
Phenylalanine hydroxylase deficiency exhibits mutation heterogeneity in two large old order Amish settlementsHeng Wang
Das Deutsch Center (DDC) Clinic for Special Needs Children, Middlefield, Ohio 44062, USA
Am J Med Genet A 143:1938-40. 2007
Homozygous mutation in SAMHD1 gene causes cerebral vasculopathy and early onset strokeBaozhong Xin
DDC Clinic for Special Needs Children, Middlefield, OH 44062, USA
Proc Natl Acad Sci U S A 108:5372-7. 2011
..The function of SAMHD1 remains unclear, but the inflammatory vasculopathies of the brain found in the patients with SAMHD1 mutation indicate its important roles in immunoregulation and cerebral vascular hemeostasis...
Homozygous frameshift mutation in TMCO1 causes a syndrome with craniofacial dysmorphism, skeletal anomalies, and mental retardationBaozhong Xin
DDC Clinic for Special Needs Children, Middlefield, OH 44062, USA
Proc Natl Acad Sci U S A 107:258-63. 2010
..This report shows a TMCO1 sequence variant being associated with a genetic disorder in human. We propose "TMCO1 defect syndrome" as the name of this condition...
Homozygosity for a novel splice site mutation in the cardiac myosin-binding protein C gene causes severe neonatal hypertrophic cardiomyopathyBaozhong Xin
Das Deutsch Center Clinic for Special Needs Children, Middlefield, Ohio 44062, USA
Am J Med Genet A 143:2662-7. 2007
..It is also concerning that many unidentified heterozygous individuals who are at risk for development of hypertrophic cardiomyopathy do not receive proper medical attention in the communities...
[Fine mapping of susceptibility genes loci within chromosome 1 in Chinese Han families with type 2 diabetes]Wei-Nan Du
National Laboratory of Medical Molecular Biology, Institute of Basic Medical Sciences, CAMS and PUMC, Beijing 100005, China
Zhongguo Yi Xue Ke Xue Yuan Xue Bao 24:234-7. 2002
..The fact that all 5 loci at the P terminal region displayed linkage with diabetes suggests that more than 1 susceptibility gene may reside in this region...
Lack of spartin protein in Troyer syndrome: a loss-of-function disease mechanism?Joanna C Bakowska
Cellular Neurology Unit, National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, Maryland 20892 3704, USA
Arch Neurol 65:520-4. 2008
..This mutation is predicted to cause premature termination of the spartin protein. However, it remains unknown whether this truncated spartin protein is absent or is present and partially functional in patients...
Cohen syndrome: report of nine cases and review of the literature, with emphasis on ophthalmic featuresMehryar Taban
Department of Pediatric Ophthalmology and the Center for Genetic Eye Diseases, Cole Eye Institute, The Cleveland Clinic Foundation, Cleveland, OH 44195, USA
J AAPOS 11:431-7. 2007
..To review the clinical features of reported cases of Cohen syndrome with a focus on ophthalmic features and report nine new cases...
Etiology of vision loss in ganglioside GM3 synthase deficiencyFahhad Farukhi
Cole Eye Institute, Cleveland Clinic Foundation, Cleveland, OH 44195, USA
Ophthalmic Genet 27:89-91. 2006
..CONCLUSIONS: Vision loss in GM3 synthase deficiency results from central nervous system and optic nerve involvement. Retinal function appears to be otherwise normal into the teenage years...
[Single nucleotide polymorphisms in CAPN10 gene of Chinese population and its correlation with type 2 diabetes mellitus in Han people of northern China]Hong xia Sun
National Laboratory of Medical Molecular Biology, Institute of Basic Medical Sciences, CAMS and PUMC, Beijing 100005, China
Zhongguo Yi Xue Ke Xue Yuan Xue Bao 24:228-33. 2002
..To investigate the distribution of the single nucleotide polymorphisms (SNPs) in CAPN10 gene in Chinese population and their relation with type 2 diabetes mellitus in Han people of Northern China...
Prolidase deficiency and the biochemical assays used in its diagnosisBiji T Kurien
Arthritis and Immunology Program, Oklahoma Medical Research Foundation, Oklahoma City, OK 73104, USA
Anal Biochem 349:165-75. 2006
Infantile-onset symptomatic epilepsy syndrome caused by a homozygous loss-of-function mutation of GM3 synthaseMichael A Simpson
Department of Medical Genetics, St George s Hospital Medical School, University of London, Cranmer Terrace, London SW17 0RE, UK
Nat Genet 36:1225-9. 2004
....
Determination of prolidase activity using matrix-assisted laser desorption/ionization time-of-flight mass spectrometryBiji T Kurien
Arthritis and Immunology Program, Oklahoma Medical Research Foundation, Oklahoma City, OK 73104, USA
Anal Biochem 331:224-9. 2004
..Using this method we have unambiguously identified subjects with homozygous or heterozygous prolidase deficiency. In addition to the advantage of rapid sample preparation time, this method is highly specific, reproducible, and sensitive...
[Linkage disequilibrium analysis of the single nucleotide polymorphisms in the PRKCZ gene]Hong xia Sun
National Laboratory of Medical Molecular Biology, Institute of Basic Medical Sciences, CAMS, PUMC, Beijing 100005, China
Zhongguo Yi Xue Ke Xue Yuan Xue Bao 24:474-80. 2002
....
[Functional analysis of the single nucleotide polymorphisms in the PRKCZ gene]Hong-Xia Sun
National Laboratory of Medical Molecular Biology, Institute of Basic Medical Sciences, CAMS, PUMC, Beijing 100005, China
Zhongguo Yi Xue Ke Xue Yuan Xue Bao 24:471-3. 2002
..Two SNPs in the gene play a role in the pathogenesis of the disease by affecting the expression level of PRKCZ gene...
[The association of two single nucleotide polymorphisms in PRKCZ and UTS2 respectively with type 2 diabetes in Han people of northern China]Hong xia Sun
National Laboratory of Medical Molecular Biology, Institute of Basic Medical Sciences, CAMS and PUMC, Beijing 100005, China
Zhongguo Yi Xue Ke Xue Yuan Xue Bao 24:223-7. 2002
..To probe the candidate susceptibility gene (s) of type 2 diabetes in the formal mapping region, 1p36.33-p36.23, in Han people of Northern China using single nucleotide polymorphisms (SNPs)...