نتایج جستجو برای: aprataxin aptx

تعداد نتایج: 112  

Journal: :Iranian biomedical journal 2012
Nayereh Nouri Narges Nouri Omid Aryani Behnam Kamalidehghan Maryam Sedghi Massoud Houshmand

BACKGROUND Ataxia with oculomotor apraxia type 1 (AOA1) shows early onset with autosomal recessive inheritance and is caused by a mutation in the aprataxin (APTX) gene encoding for the APTX protein. METHODS In this study, a 7-year-old girl born of a first-cousin consanguineous marriage was described with early-onset progressive ataxia and AOA, with increased cholesterol concentration and decr...

Behnam Kamalidehghan, Maryam Sedghi, Massoud Houshmand, Narges Nouri, Nayereh Nouri, Omid Aryani,

Background: Ataxia with oculomotor apraxia type 1 (AOA1) shows early onset with autosomal recessive inheritance and is caused by a mutation in the aprataxin (APTX) gene encoding for the APTX protein. Methods: In this study, a 7-year-old girl born of a first-cousin consanguineous marriage was described with early-onset progressive ataxia and AOA, with increased cholesterol concentration and decr...

Journal: :iranian biomedical journal 0
نیره نوری nayereh nouri نرگس نوری narges nouri امید آریانی omid aryani بهنام کمالی دهقان behnam kamalidehghan مریم صدقی maryam sedghi مسعود هوشمند massoud houshmand

background: ataxia with oculomotor apraxia type 1 (aoa1) shows early onset with autosomal recessive inheritance and is caused by a mutation in the aprataxin (aptx) gene encoding for the aptx protein. methods: in this study, a 7-year-old girl born of a first-cousin consanguineous marriage was described with early-onset progressive ataxia and aoa, with increased cholesterol concentration and decr...

2017
Humera Manzoor Ihtisham Bukhari Muhammad Wajid Yuanwei Zhang Huan Zhang Norbert Brüggemann Christine Klein Qinghua Shi Sadaf Naz

Dear Editor, Ataxia with oculomotor apraxia type 1 (AOA1, MIM 208920) is an autosomal recessive disorder characterized by early-onset cerebellar ataxia, polyneuropathy, oculomotor apraxia, hypoalbuminemia, and hypercholesterolemia.1 It is caused by pathogenic variants of APTX, which encodes the aprataxin protein that is involved in DNA strand-break repair.1 We recruited a consanguineous family ...

Journal: :Proceedings of the National Academy of Sciences of the United States of America 2006
Makito Hirano Yoshiko Furiya Hirohide Asai Akira Yasui Satoshi Ueno

Triple A syndrome is an autosomal recessive neuroendocrinological disease caused by mutations in a gene that encodes 546 amino acid residues. The encoded protein is the nucleoporin ALADIN, a component of nuclear pore complex (NPC). We identified a mutant ALADIN(I482S) that fails to target NPC and investigated the consequences of mistargeting using cultured fibroblasts (I482Sf) from a patient wi...

Journal: :Blood 2008
Mitch Raponi Jeffrey E Lancet Hongtao Fan Lesley Dossey Grace Lee Ivana Gojo Eric J Feldman Jason Gotlib Lawrence E Morris Peter L Greenberg John J Wright Jean-Luc Harousseau Bob Löwenberg Richard M Stone Peter De Porre Yixin Wang Judith E Karp

At present, there is no method available to predict response to farnesyltransferase inhibitors (FTIs). We analyzed gene expression profiles from the bone marrow of patients from a phase 2 study of the FTI tipifarnib in older adults with previously untreated acute myeloid leukemia (AML). The RASGRP1/APTX gene expression ratio was found to predict response to tipifarnib with the greatest accuracy...

Journal: :Journal of neurological disorders & stroke 2013
Catarina M Quinzii Michio Hirano Ali Naini

In 2001, we described six patients with cerebellar ataxia and severe deficiency of coenzyme Q10 (CoQ10, ubiquinone) in skeletal muscle [1]. Within one year, we described 13 additional patients [2]; therefore, we suspected this was not a very rare syndrome. Twelve years after our original report, cerebellar ataxia and atrophy has emerged as the most common clinical presentation of CoQ10 deficien...

Journal: :Blood 2012
Judith E Karp Tatiana I Vener Mitch Raponi Ellen K Ritchie B Douglas Smith Steven D Gore Lawrence E Morris Eric J Feldman Jacqueline M Greer Sami Malek Hetty E Carraway Valerie Ironside Steven Galkin Mark J Levis Michael A McDevitt Gail R Roboz Christopher D Gocke Carlo Derecho John Palma Yixin Wang Scott H Kaufmann John J Wright Elizabeth Garret-Mayer

Tipifarnib (T) exhibits modest activity in elderly adults with newly diagnosed acute myelogenous leukemia (AML). Based on preclinical synergy, a phase 1 trial of T plus etoposide (E) yielded 25% complete remission (CR). We selected 2 comparable dose levels for a randomized phase 2 trial in 84 adults (age range, 70-90 years; median, 76 years) who were not candidates for conventional chemotherapy...

2014
Martin Meagher Robert N. Lightowlers

In recent years, our knowledge surrounding mammalian mitochondrial DNA (mtDNA) damage and repair has increased significantly. Greater insights into the factors that govern mtDNA repair are being elucidated, thus contributing to an increase in our understanding year on year. In this short review two enzymes, tyrosyl-DNA-phosphodiesterase 1 (TDP1) and aprataxin (APTX), involved in mitochondrial s...

2013
Guofeng Yue Jia Wei Xiaoping Qian Lixia Yu Zhengyun Zou Wenxian Guan Hao Wang Jie Shen Baorui Liu

OBJECTIVE The present study was designed to examine the anticancer effect of Traditional Chinese Medicine of polyphyllin I (PPI) and evodiamine (EVO) on freshly-removed gastric tumor tissues. METHODS Sixty freshly-removed gastric tumor tissues were collected. Their sensitivity to PPI, EVO, platinum (Pt), 5-FU, irinotecan (CPT-11) were determined by histoculture drug response assay (HDRA). Tho...

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