1 |
11/01/2007 |
17554762 |
Centrosomal-ciliary gene CEP290/NPHP6 mutations result in blindness with unexpected sparing of photoreceptors and visual brain: implications for therapy of Leber congenital amaurosis. |
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Mutations in the centrosomal-ciliary gene CEP290/NPHP6 are associated with Joubert syndrome and are the most common cause of the childhood recessive blindness known as Leber congenital amaurosis (LCA)...
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2 |
10/25/2007 |
17964524 |
Leber Congenital Amaurosis-A Model for Efficient Genetic Testing of Heterogeneous Disorders: LXIV Edward Jackson Memorial Lecture. |
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PURPOSE: To critically evaluate our experience in molecular testing of Leber congenital amaurosis (LCA) and to use this information to devise a general approach to heterogeneous recessive disorders. C...
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3 |
10/24/2007 |
17960139 |
Mutational analysis of the RPGRIP1L gene in patients with Joubert syndrome and nephronophthisis. |
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Joubert syndrome (JS) is an autosomal recessive disorder, consisting of mental retardation, cerebellar vermis aplasia, an irregular breathing pattern, and retinal degeneration. Nephronophthisis (NPHP)...
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4 |
10/06/2007 |
18054307 |
DNA analysis of AHI1, NPHP1 and CYCLIN D1 in Joubert syndrome patients from the Netherlands. |
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Joubert syndrome (JBS) is a clinically variable and genetically heterogeneous developmental brain disorder with autosomal recessive inheritance. Five genes, AHI1, NPHP1, CEP290, MKS3, and RPGRIP1L, an...
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5 |
10/02/2007 |
17898177 |
Hypomorphic CEP290/NPHP6 mutations result in anosmia caused by the selective loss of G proteins in cilia of olfactory sensory neurons. |
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Cilia regulate diverse functions such as motility, fluid balance, and sensory perception. The cilia of olfactory sensory neurons (OSNs) compartmentalize the signaling proteins necessary for odor detec...
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6 |
10/01/2007 |
17617513 |
Mutation analysis of NPHP6/CEP290 in patients with Joubert syndrome and Senior-Løken syndrome. |
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BACKGROUND: Nephronophthisis (NPHP) is an autosomal recessive cystic kidney disease that constitutes the most common genetic cause of renal failure in the first three decades of life. Using positional...
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7 |
09/01/2007 |
17724218 |
Clinical and molecular genetics of Leber's congenital amaurosis: a multicenter study of Italian patients. |
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PURPOSE: To identify the molecular basis of Leber's congenital amaurosis (LCA) in a cohort of Italian patients and to perform genotype-phenotype analysis. METHODS: DNA samples from 95 patients with LC...
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8 |
08/17/2007 |
17705300 |
Mutations of the CEP290 gene encoding a centrosomal protein cause Meckel-Gruber syndrome. |
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Meckel-Gruber syndrome (MKS) is an autosomal recessive, lethal multisystemic disorder characterized by meningooccipital encephalocele, cystic kidney dysplasia, hepatobiliary ductal plate malformation,...
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9 |
07/01/2007 |
17564974 |
Pleiotropic effects of CEP290 (NPHP6) mutations extend to Meckel syndrome. |
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Meckel syndrome (MKS) is a rare autosomal recessive lethal condition characterized by central nervous system malformations, polydactyly, multicystic kidney dysplasia, and ductal changes of the liver. ...
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10 |
07/01/2007 |
17564967 |
CEP290 mutations are frequently identified in the oculo-renal form of Joubert syndrome-related disorders. |
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Joubert syndrome-related disorders (JSRDs) are a group of clinically and genetically heterogeneous conditions that share a midbrain-hindbrain malformation, the molar tooth sign (MTS) visible on brain ...
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11 |
07/01/2007 |
17558409 |
The ciliary gene RPGRIP1L is mutated in cerebello-oculo-renal syndrome (Joubert syndrome type B) and Meckel syndrome. |
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Cerebello-oculo-renal syndrome (CORS), also called Joubert syndrome type B, and Meckel (MKS) syndrome belong to the group of developmental autosomal recessive disorders that are associated with primar...
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12 |
05/01/2007 |
17507457 |
Mutation in CEP290 discovered for cat model of human retinal degeneration. |
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A mutation in the CEP290 gene is reported in a cat pedigree segregating for autosomal recessive (AR) late-onset photoreceptor degeneration (rdAc). An initial screen of 39 candidate genes and genomic l...
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13 |
05/01/2007 |
17409309 |
High NPHP1 and NPHP6 mutation rate in patients with Joubert syndrome and nephronophthisis: potential epistatic effect of NPHP6 and AHI1 mutations in patients with NPHP1 mutations. |
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Joubert syndrome (JS) is an autosomal recessive disorder that is described in patients with cerebellar ataxia, mental retardation, hypotonia, and neonatal respiratory dysregulation. Kidney involvement...
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14 |
05/01/2007 |
17377524 |
Joubert syndrome (and related disorders) (OMIM 213300). |
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Joubert syndrome (JS) and related disorders are characterized by the 'molar tooth sign' (cerebellar vermis hypoplasia and brainstem anomalies) on MRI, hypotonia, developmental delay, ataxia, irregular...
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15 |
04/01/2007 |
17345604 |
Spectrum of NPHP6/CEP290 mutations in Leber congenital amaurosis and delineation of the associated phenotype. |
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Leber congenital amaurosis (LCA) is the earliest and most severe retinal degeneration responsible for congenital blindness. Hitherto, 13 LCA genes have been mapped, nine of which have been identified....
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16 |
01/01/2007 |
18079693 |
Frequency of CEP290 c.2991_1655A>G mutation in 175 Spanish families affected with Leber congenital amaurosis and early-onset retinitis pigmentosa. |
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PURPOSE: Leber congenital amaurosis (LCA) is the most severe inherited retinopathy with the earliest age of onset. To date, eleven genes have been reported to cause the non-syndromic LCA phenotype. Th...
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17 |
01/01/2007 |
17160906 |
The Meckel-Gruber syndrome gene, MKS3, is mutated in Joubert syndrome. |
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Joubert syndrome (JS) is an autosomal recessive disorder characterized by cerebellar vermis hypoplasia associated with hypotonia, developmental delay, abnormal respiratory patterns, and abnormal eye m...
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18 |
12/01/2006 |
17148037 |
Lumpers or splitters? The role of molecular diagnosis in Leber congenital amaurosis. |
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Clarification and classification of the congenital form of blindness known as Leber congenital amaurosis (LCA) continues to provide its challenges and dilemmas. Until recently, seven genes have been i...
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19 |
09/01/2006 |
16909394 |
Mutations in the CEP290 (NPHP6) gene are a frequent cause of Leber congenital amaurosis. |
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Leber congenital amaurosis (LCA) is one of the main causes of childhood blindness. To date, mutations in eight genes have been described, which together account for approximately 45% of LCA cases. We ...
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20 |
06/01/2006 |
16682973 |
The centrosomal protein nephrocystin-6 is mutated in Joubert syndrome and activates transcription factor ATF4. |
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The molecular basis of nephronophthisis, the most frequent genetic cause of renal failure in children and young adults, and its association with retinal degeneration and cerebellar vermis aplasia in J...
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