نتایج جستجو برای: frataxin

تعداد نتایج: 673  

2013
Heather L. Plasterer Eric C. Deutsch Matthew Belmonte Elizabeth Egan David R. Lynch James R. Rusche

BACKGROUND Friedreich ataxia is a progressive neurodegenerative disorder caused by GAA triplet repeat expansions or point mutations in the FXN gene and, ultimately, a deficiency in the levels of functional frataxin protein. Heterozygous carriers of the expansion express approximately 50% of normal frataxin levels yet manifest no clinical symptoms, suggesting that therapeutic approaches that inc...

2015
Alessandra Rufini Francesca Cavallo Ivano Condò Silvia Fortuni Gabriella De Martino Ottaviano Incani Almerinda Di Venere Monica Benini Damiano Sergio Massaro Gaetano Arcuri Dario Serio Florence Malisan Roberto Testi

Friedreich ataxia is an inherited neurodegenerative disease that leads to progressive disability. There is currently no effective treatment and patients die prematurely. The underlying genetic defect leads to reduced expression of the mitochondrial protein frataxin. Frataxin insufficiency causes mitochondrial dysfunction and ultimately cell death, particularly in peripheral sensory ganglia. The...

Journal: :Human molecular genetics 2015
Fabio Cherubini Dario Serio Ilaria Guccini Silvia Fortuni Gaetano Arcuri Ivano Condò Alessandra Rufini Shadman Moiz Serena Camerini Marco Crescenzi Roberto Testi Florence Malisan

Defective expression of frataxin is responsible for the inherited, progressive degenerative disease Friedreich's Ataxia (FRDA). There is currently no effective approved treatment for FRDA and patients die prematurely. Defective frataxin expression causes critical metabolic changes, including redox imbalance and ATP deficiency. As these alterations are known to regulate the tyrosine kinase Src, ...

2017
Monica Benini Silvia Fortuni Ivano Condò Giulia Alfedi Florence Malisan Nicola Toschi Dario Serio Damiano Sergio Massaro Gaetano Arcuri Roberto Testi Alessandra Rufini

Friedreich ataxia (FRDA) is a severe genetic neurodegenerative disease caused by reduced expression of the mitochondrial protein frataxin. To date, there is no therapy to treat this condition. The amount of residual frataxin critically affects the severity of the disease; thus, attempts to restore physiological frataxin levels are considered therapeutically relevant. Frataxin levels are control...

2002
Erika M. Becker Judith M. Greer Prem Ponka

Friedreich ataxia (FA) is caused by decreased frataxin expression that results in mitochondrial iron (Fe) overload. However, the role of frataxin in mammalian Fe metabolism remains unclear. In this investigation we examined the function of frataxin in Fe metabolism by implementing a well-characterized model of erythroid differentiation, namely, Friend cells induced using dimethyl sulfoxide (DMS...

Journal: :Human molecular genetics 2002
Patrizia Cavadini Heather A O'Neill Oldrich Benada Grazia Isaya

Friedreich ataxia (FRDA) is an autosomal recessive degenerative disease caused by a deficiency of frataxin, a conserved mitochondrial protein of unknown function. Mitochondrial iron accumulation, loss of iron-sulfur cluster-containing enzymes and increased oxidative damage occur in yeast and mouse frataxin-depleted mutants as well as tissues and cell lines from FRDA patients, suggesting that fr...

2014
Barbara Carletti Emanuela Piermarini Giulia Tozzi Lorena Travaglini Alessandra Torraco Anna Pastore Marco Sparaco Sara Petrillo Rosalba Carrozzo Enrico Bertini Fiorella Piemonte

Friedreich's ataxia (FRDA) is a hereditary neurodegenerative disease characterized by a reduced synthesis of the mitochondrial iron chaperon protein frataxin as a result of a large GAA triplet-repeat expansion within the first intron of the frataxin gene. Despite neurodegeneration being the prominent feature of this pathology involving both the central and the peripheral nervous system, informa...

2015
Michael Lazaropoulos Yina Dong Elisia Clark Nathaniel R Greeley Lauren A Seyer Karlla W Brigatti Carlton Christie Susan L Perlman George R Wilmot Christoper M Gomez Katherine D Mathews Grace Yoon Theresa Zesiewicz Chad Hoyle Sub H Subramony Alicia F Brocht Jennifer M Farmer Robert B Wilson Eric C Deutsch David R Lynch

OBJECTIVE Friedreich ataxia (FRDA) is an autosomal recessive ataxia resulting from mutations in the frataxin gene (FXN). Such mutations, usually expanded guanine-adenine-adenine (GAA) repeats, give rise to decreased levels of frataxin protein in both affected and unaffected tissues. The goal was to understand the relationship of frataxin levels in peripheral tissues to disease status. METHODS...

2010
René Thierbach Gunnar Drewes Markus Fusser Anja Voigt Doreen Kuhlow Urte Blume Tim J. Schulz Carina Reiche Hansruedi Glatt Bernd Epe Pablo Steinberg Michael Ristow

DNA-repair mechanisms enable cells to maintain their genetic information by protecting it from mutations that may cause malignant growth. Recent evidence suggests that specific DNA-repair enzymes contain ISCs (iron-sulfur clusters). The nuclearencoded protein frataxin is essential for the mitochondrial biosynthesis of ISCs. Frataxin deficiency causes a neurodegenerative disorder named Friedreic...

Journal: :Human molecular genetics 2006
Oleksandr Gakh Sungjo Park Gang Liu Lee Macomber James A Imlay Gloria C Ferreira Grazia Isaya

Friedreich ataxia is a severe autosomal-recessive disease characterized by neurodegeneration, cardiomyopathy and diabetes, resulting from reduced synthesis of the mitochondrial protein frataxin. Although frataxin is ubiquitously expressed, frataxin deficiency leads to a selective loss of dorsal root ganglia neurons, cardiomyocytes and pancreatic beta cells. How frataxin normally promotes surviv...

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