Double-blind therapeutic trial in Angelman syndrome using betaine and folic acid.

PubWeight™: 0.96‹?› | Rank: Top 15%

🔗 View Article (PMC 3172130)

Published in Am J Med Genet A on August 01, 2010

Authors

Sarika U Peters1, Lynne M Bird, Virginia Kimonis, Daniel G Glaze, Lina M Shinawi, Terry Jo Bichell, Rene Barbieri-Welge, Mark Nespeca, Irina Anselm, Susan Waisbren, Erica Sanborn, Qin Sun, William E O'Brien, Arthur L Beaudet, Carlos A Bacino

Author Affiliations

1: Department of Molecular and Human Genetics, Baylor College of Medicine, Houston, Texas, USA.

Articles cited by this

UBE3A/E6-AP mutations cause Angelman syndrome. Nat Genet (1997) 5.97

Folate levels and neural tube defects. Implications for prevention. JAMA (1995) 5.42

Maternal methyl supplements in mice affect epigenetic variation and DNA methylation of offspring. J Nutr (2002) 4.97

Mutation of the Angelman ubiquitin ligase in mice causes increased cytoplasmic p53 and deficits of contextual learning and long-term potentiation. Neuron (1998) 4.92

De novo truncating mutations in E6-AP ubiquitin-protein ligase gene (UBE3A) in Angelman syndrome. Nat Genet (1997) 4.65

Maternal epigenetics and methyl supplements affect agouti gene expression in Avy/a mice. FASEB J (1998) 4.62

The IC-SNURF-SNRPN transcript serves as a host for multiple small nucleolar RNA species and as an antisense RNA for UBE3A. Hum Mol Genet (2001) 3.07

Angelman syndrome 2005: updated consensus for diagnostic criteria. Am J Med Genet A (2006) 2.86

The Angelman syndrome candidate gene, UBE3A/E6-AP, is imprinted in brain. Nat Genet (1997) 2.77

The Angelman syndrome ubiquitin ligase localizes to the synapse and nucleus, and maternal deficiency results in abnormal dendritic spine morphology. Hum Mol Genet (2007) 2.60

Folate treatment and unbalanced methylation and changes of allelic expression induced by hyperhomocysteinaemia in patients with uraemia. Lancet (2003) 2.39

Moderate folate depletion increases plasma homocysteine and decreases lymphocyte DNA methylation in postmenopausal women. J Nutr (1998) 2.36

Angelman syndrome: consensus for diagnostic criteria. Angelman Syndrome Foundation. Am J Med Genet (1995) 2.34

An imprinted antisense RNA overlaps UBE3A and a second maternally expressed transcript. Nat Genet (1998) 2.16

Imprinting of the Angelman syndrome gene, UBE3A, is restricted to brain. Nat Genet (1997) 1.92

Sporadic imprinting defects in Prader-Willi syndrome and Angelman syndrome: implications for imprint-switch models, genetic counseling, and prenatal diagnosis. Am J Hum Genet (1998) 1.87

Neurobehavioral and electroencephalographic abnormalities in Ube3a maternal-deficient mice. Neurobiol Dis (2002) 1.84

A mixed epigenetic/genetic model for oligogenic inheritance of autism with a limited role for UBE3A. Am J Med Genet A (2004) 1.79

Molecular characterization of two proximal deletion breakpoint regions in both Prader-Willi and Angelman syndrome patients. Am J Hum Genet (1995) 1.78

Predominant maternal expression of the mouse Atp10c in hippocampus and olfactory bulb. J Hum Genet (2003) 1.72

A survey of knowledge and use of folic acid among women of child-bearing age in Dublin. J Public Health Med (1997) 1.72

Genetic counseling in Angelman syndrome: the challenges of multiple causes. Am J Med Genet (1998) 1.71

Betaine in the treatment of homocystinuria due to 5,10-methylenetetrahydrofolate reductase deficiency. Eur J Pediatr (1984) 1.47

Genetics of Angelman syndrome. Am J Hum Genet (1999) 1.43

Autism in Angelman syndrome: implications for autism research. Clin Genet (2004) 1.37

Autism in Angelman syndrome: a population-based study. Pediatr Neurol (1996) 1.34

Chromosome 15 uniparental disomy is not frequent in Angelman syndrome. Am J Hum Genet (1991) 1.30

The Angelman ("happy puppet") syndrome. Am J Med Genet (1982) 1.27

Maternal but not paternal transmission of 15q11-13-linked nondeletion Angelman syndrome leads to phenotypic expression. Nat Genet (1992) 1.25

Imprinting in Angelman and Prader-Willi syndromes. Curr Opin Genet Dev (1998) 1.23

Paternal uniparental disomy in a child with a balanced 15;15 translocation and Angelman syndrome. Am J Med Genet (1993) 1.17

Angelman syndrome. Curr Probl Pediatr (1995) 1.09

Clinical spectrum and molecular diagnosis of Angelman and Prader-Willi syndrome patients with an imprinting mutation. Am J Med Genet (1997) 1.07

Strategies for the treatment of cystathionine beta-synthase deficiency: the experience of the Willink Biochemical Genetics Unit over the past 30 years. Eur J Pediatr (1998) 1.02

Folic acid and neural tube defect: can't we come to closure? Am J Public Health (1992) 1.01

Angelman syndrome: clinical profile. J Child Neurol (1992) 0.99

Paternal uniparental disomy of chromosome 15 in a child with Angelman syndrome. Ann Neurol (1992) 0.98

Progressive cerebral edema associated with high methionine levels and betaine therapy in a patient with cystathionine beta-synthase (CBS) deficiency. Am J Med Genet (2002) 0.97

Betaine for treatment of homocystinuria caused by methylenetetrahydrofolate reductase deficiency. Arch Dis Child (1989) 0.93

Reassessing folic acid consumption patterns in the United States (1999 2004): potential effect on neural tube defects and overexposure to folate. Am J Clin Nutr (2007) 0.92

Betaine dose and treatment intervals in therapy for homocystinuria due to 5,10-methylenetetrahydrofolate reductase deficiency. J Inherit Metab Dis (1998) 0.88

Structure and function of the human chromosome 15 imprinting center. J Cell Physiol (1997) 0.86

Methylation demand and homocysteine metabolism. Adv Enzyme Regul (2004) 0.86

A study of the treatment of Rett syndrome with folate and betaine. J Child Neurol (2009) 0.84

Genomic imprinting: significance in development and diseases and the molecular mechanisms. J Biochem (1996) 0.84

Effect of betaine on S-adenosylmethionine levels in the cerebrospinal fluid in a patient with methylenetetrahydrofolate reductase deficiency and peripheral neuropathy. J Inherit Metab Dis (1994) 0.83

Methylenetetrahydrofolate reductase deficiency in a patient with phenotypic findings of Angelman syndrome. Am J Med Genet (1998) 0.83

Implementation and outcomes of recommended folic acid supplementation in Mexican-American women with prior neural tube defect-affected pregnancies. Prev Med (2005) 0.80

Imprinting mutations on human chromosome 15. Hum Mutat (1997) 0.79

Articles by these authors

Clinical whole-exome sequencing for the diagnosis of mendelian disorders. N Engl J Med (2013) 15.85

The NIH Roadmap Epigenomics Mapping Consortium. Nat Biotechnol (2010) 13.99

Multiple recurrent de novo CNVs, including duplications of the 7q11.23 Williams syndrome region, are strongly associated with autism. Neuron (2011) 10.61

Chromosomal microarray versus karyotyping for prenatal diagnosis. N Engl J Med (2012) 4.97

Recurrent reciprocal 1q21.1 deletions and duplications associated with microcephaly or macrocephaly and developmental and behavioral abnormalities. Nat Genet (2008) 4.87

Prader-Willi phenotype caused by paternal deficiency for the HBII-85 C/D box small nucleolar RNA cluster. Nat Genet (2008) 4.10

Rett syndrome: revised diagnostic criteria and nomenclature. Ann Neurol (2010) 4.03

Genetic dissection of complex traits with chromosome substitution strains of mice. Science (2004) 3.57

Clinical implementation of chromosomal microarray analysis: summary of 2513 postnatal cases. PLoS One (2007) 3.53

Using VAAST to identify an X-linked disorder resulting in lethality in male infants due to N-terminal acetyltransferase deficiency. Am J Hum Genet (2011) 3.43

Development and validation of a CGH microarray for clinical cytogenetic diagnosis. Genet Med (2005) 3.39

Recurrent reciprocal 16p11.2 rearrangements associated with global developmental delay, behavioural problems, dysmorphism, epilepsy, and abnormal head size. J Med Genet (2009) 3.33

Use of array CGH in the evaluation of dysmorphology, malformations, developmental delay, and idiopathic mental retardation. Curr Opin Genet Dev (2007) 3.08

Chromosome catastrophes involve replication mechanisms generating complex genomic rearrangements. Cell (2011) 3.03

VCP/p97 is essential for maturation of ubiquitin-containing autophagosomes and this function is impaired by mutations that cause IBMPFD. Autophagy (2010) 2.99

Mutations in the gene encoding filamin B disrupt vertebral segmentation, joint formation and skeletogenesis. Nat Genet (2004) 2.94

Detection of clinically relevant exonic copy-number changes by array CGH. Hum Mutat (2010) 2.93

Angelman syndrome 2005: updated consensus for diagnostic criteria. Am J Med Genet A (2006) 2.86

Clinical and molecular phenotype of Aicardi-Goutieres syndrome. Am J Hum Genet (2007) 2.81

Increased MECP2 gene copy number as the result of genomic duplication in neurodevelopmentally delayed males. Genet Med (2006) 2.78

Epigenetics and human disease. Annu Rev Genomics Hum Genet (2004) 2.78

Mutations in ADAR1 cause Aicardi-Goutières syndrome associated with a type I interferon signature. Nat Genet (2012) 2.63

The Angelman syndrome ubiquitin ligase localizes to the synapse and nucleus, and maternal deficiency results in abnormal dendritic spine morphology. Hum Mol Genet (2007) 2.60

Complex rearrangements in patients with duplications of MECP2 can occur by fork stalling and template switching. Hum Mol Genet (2009) 2.50

Evaluation, diagnosis, and treatment of gastrointestinal disorders in individuals with ASDs: a consensus report. Pediatrics (2010) 2.45

Increased LIS1 expression affects human and mouse brain development. Nat Genet (2009) 2.45

Genetic heterogeneity in Rubinstein-Taybi syndrome: mutations in both the CBP and EP300 genes cause disease. Am J Hum Genet (2005) 2.40

Synaptic dysfunction and abnormal behaviors in mice lacking major isoforms of Shank3. Hum Mol Genet (2011) 2.38

Bacterial artificial chromosome-emulation oligonucleotide arrays for targeted clinical array-comparative genomic hybridization analyses. Genet Med (2008) 2.36

Acute toxicity after high-dose systemic injection of helper-dependent adenoviral vectors into nonhuman primates. Hum Gene Ther (2004) 2.32

Spectrum of CHD7 mutations in 110 individuals with CHARGE syndrome and genotype-phenotype correlation. Am J Hum Genet (2005) 2.22

An expanded version of the Hammersmith Functional Motor Scale for SMA II and III patients. Neuromuscul Disord (2007) 2.17

Physical map of 1p36, placement of breakpoints in monosomy 1p36, and clinical characterization of the syndrome. Am J Hum Genet (2003) 2.15

A genome-wide association study identifies susceptibility loci for nonsyndromic sagittal craniosynostosis near BMP2 and within BBS9. Nat Genet (2012) 2.12

Nutritional phases in Prader-Willi syndrome. Am J Med Genet A (2011) 2.08

Establishing a consortium for the study of rare diseases: The Urea Cycle Disorders Consortium. Mol Genet Metab (2010) 2.07

Autism and other neuropsychiatric symptoms are prevalent in individuals with MeCP2 duplication syndrome. Ann Neurol (2009) 2.07

Mutations in the gene encoding the calcium-permeable ion channel TRPV4 produce spondylometaphyseal dysplasia, Kozlowski type and metatropic dysplasia. Am J Hum Genet (2009) 2.02

Genomic imbalances in neonates with birth defects: high detection rates by using chromosomal microarray analysis. Pediatrics (2008) 2.00

Speech delay and autism spectrum behaviors are frequently associated with duplication of the 7q11.23 Williams-Beuren syndrome region. Genet Med (2007) 1.98

A small recurrent deletion within 15q13.3 is associated with a range of neurodevelopmental phenotypes. Nat Genet (2009) 1.98

Mutant valosin-containing protein causes a novel type of frontotemporal dementia. Ann Neurol (2005) 1.94

Derangements of hippocampal calcium/calmodulin-dependent protein kinase II in a mouse model for Angelman mental retardation syndrome. J Neurosci (2003) 1.93

Control of leukocyte rolling velocity in TNF-alpha-induced inflammation by LFA-1 and Mac-1. Blood (2002) 1.92

Mouse model for human arginase deficiency. Mol Cell Biol (2002) 1.88

Molecular analysis expands the spectrum of phenotypes associated with GLI3 mutations. Hum Mutat (2010) 1.88

Microarray-based CGH detects chromosomal mosaicism not revealed by conventional cytogenetics. Am J Med Genet A (2007) 1.85

A common X-linked inborn error of carnitine biosynthesis may be a risk factor for nondysmorphic autism. Proc Natl Acad Sci U S A (2012) 1.85

Loss of MeCP2 in aminergic neurons causes cell-autonomous defects in neurotransmitter synthesis and specific behavioral abnormalities. Proc Natl Acad Sci U S A (2009) 1.79

A mixed epigenetic/genetic model for oligogenic inheritance of autism with a limited role for UBE3A. Am J Med Genet A (2004) 1.79

Clinical use of array comparative genomic hybridization (aCGH) for prenatal diagnosis in 300 cases. Prenat Diagn (2009) 1.78

Truncating mutations of MAGEL2 cause Prader-Willi phenotypes and autism. Nat Genet (2013) 1.76

Lethal toxicity, severe endothelial injury, and a threshold effect with high doses of an adenoviral vector in baboons. Hum Gene Ther (2002) 1.69

Pathological consequences of VCP mutations on human striated muscle. Brain (2006) 1.68

Structures and molecular mechanisms for common 15q13.3 microduplications involving CHRNA7: benign or pathological? Hum Mutat (2010) 1.66

Prenatal diagnosis of chromosomal abnormalities using array-based comparative genomic hybridization. Genet Med (2006) 1.63

Methyl-CpG-binding protein 2 (MECP2) mutation type is associated with disease severity in Rett syndrome. J Med Genet (2014) 1.58

Rett syndrome: North American database. J Child Neurol (2007) 1.54

Requirement of argininosuccinate lyase for systemic nitric oxide production. Nat Med (2011) 1.54

Epigenetic profiling at mouse imprinted gene clusters reveals novel epigenetic and genetic features at differentially methylated regions. Genome Res (2009) 1.52

Identification of novel deletions of 15q11q13 in Angelman syndrome by array-CGH: molecular characterization and genotype-phenotype correlations. Eur J Hum Genet (2007) 1.52

Identification of chromosome abnormalities in subtelomeric regions by microarray analysis: a study of 5,380 cases. Am J Med Genet A (2008) 1.51

Pseudo-hydrodynamic delivery of helper-dependent adenoviral vectors into non-human primates for liver-directed gene therapy. Mol Ther (2007) 1.50

Effect of alternative pathway therapy on branched chain amino acid metabolism in urea cycle disorder patients. Mol Genet Metab (2004) 1.50

Creatine metabolism in combined methylmalonic aciduria and homocystinuria. Ann Neurol (2005) 1.46

An integrated approach to the diagnosis and prospective management of partial ornithine transcarbamylase deficiency. Pediatrics (2002) 1.43

Development of a comparative genomic hybridization microarray and demonstration of its utility with 25 well-characterized 1p36 deletions. Hum Mol Genet (2003) 1.42

Copy number and SNP arrays in clinical diagnostics. Annu Rev Genomics Hum Genet (2011) 1.41

Efficient, long-term hepatic gene transfer using clinically relevant HDAd doses by balloon occlusion catheter delivery in nonhuman primates. Mol Ther (2008) 1.40

Identification of ANKRD11 and ZNF778 as candidate genes for autism and variable cognitive impairment in the novel 16q24.3 microdeletion syndrome. Eur J Hum Genet (2009) 1.40

Increased hepatic transduction with reduced systemic dissemination and proinflammatory cytokines following hydrodynamic injection of helper-dependent adenoviral vectors. Mol Ther (2005) 1.40

Polymicrogyria and deletion 22q11.2 syndrome: window to the etiology of a common cortical malformation. Am J Med Genet A (2006) 1.38

Altered ultrasonic vocalization and impaired learning and memory in Angelman syndrome mouse model with a large maternal deletion from Ube3a to Gabrb3. PLoS One (2010) 1.37

The Maternal Phenylketonuria International Study: 1984-2002. Pediatrics (2003) 1.37

Lifelong elimination of hyperbilirubinemia in the Gunn rat with a single injection of helper-dependent adenoviral vector. Proc Natl Acad Sci U S A (2005) 1.36

Array-based comparative genomic hybridization analysis of recurrent chromosome 15q rearrangements. Am J Med Genet A (2005) 1.35

Molecular diagnosis of Duchenne/Becker muscular dystrophy: enhanced detection of dystrophin gene rearrangements by oligonucleotide array-comparative genomic hybridization. Hum Mutat (2008) 1.35

Incidental copy-number variants identified by routine genome testing in a clinical population. Genet Med (2012) 1.33

Tissue-specific variation of Ube3a protein expression in rodents and in a mouse model of Angelman syndrome. Neurobiol Dis (2010) 1.33

Measuring the work function of carbon nanotubes with thermionic method. Nano Lett (2008) 1.33

Rett syndrome diagnostic criteria: lessons from the Natural History Study. Ann Neurol (2010) 1.33

Sustained phenotypic correction of canine hemophilia B after systemic administration of helper-dependent adenoviral vector. Hum Gene Ther (2005) 1.32

A modern approach to the treatment of mitochondrial disease. Curr Treat Options Neurol (2009) 1.32

Intercellular adhesion molecule-1 expression is required on multiple cell types for the development of experimental autoimmune encephalomyelitis. J Immunol (2007) 1.31

Insertional translocation detected using FISH confirmation of array-comparative genomic hybridization (aCGH) results. Am J Med Genet A (2010) 1.31

Human disorders of ubiquitination and proteasomal degradation. Curr Opin Pediatr (2004) 1.31

Microarray analysis for constitutional cytogenetic abnormalities. Genet Med (2007) 1.30

Deficiency of Rbbp1/Arid4a and Rbbp1l1/Arid4b alters epigenetic modifications and suppresses an imprinting defect in the PWS/AS domain. Genes Dev (2006) 1.27