Literature DB >> 30905359

Therapeutic Prospects for Friedreich's Ataxia.

Siyuan Zhang1, Marek Napierala1, Jill S Napierala2.   

Abstract

Friedreich's ataxia (FRDA) is a progressive disease affecting multiple organs that is caused by systemic insufficiency of the mitochondrial protein frataxin. Current therapeutic strategies aim to elevate frataxin levels and/or alleviate the consequences of frataxin deficiency. Recent significant advances in the FRDA therapeutic pipeline are bringing patients closer to a cure.
Copyright © 2019 Elsevier Ltd. All rights reserved.

Entities:  

Keywords:  Friedreich’s ataxia; frataxin; gene therapy; mitochondria; oligonucleotide; oxidative stress

Mesh：

Substances：

Year:  2019        PMID： 30905359      PMCID： PMC6826337          DOI： 10.1016/j.tips.2019.02.001

Source DB:  PubMed          Journal:  Trends Pharmacol Sci        ISSN： 0165-6147            Impact factor:   14.819


  18 in total

1.  Rapid and Complete Reversal of Sensory Ataxia by Gene Therapy in a Novel Model of Friedreich Ataxia.

Authors:  Françoise Piguet; Charline de Montigny; Nadège Vaucamps; Laurence Reutenauer; Aurélie Eisenmann; Hélène Puccio
Journal:  Mol Ther       Date:  2018-05-28       Impact factor: 11.454

Review 2.  Gene therapy for neurological disorders: progress and prospects.

Authors:  Benjamin E Deverman; Bernard M Ravina; Krystof S Bankiewicz; Steven M Paul; Dinah W Y Sah
Journal:  Nat Rev Drug Discov       Date:  2018-08-10       Impact factor: 84.694

Review 3.  An overview of the chemistry and biology of reactive aldehydes.

Authors:  Kristofer S Fritz; Dennis R Petersen
Journal:  Free Radic Biol Med       Date:  2012-06-28       Impact factor: 7.376

4.  Prevention and reversal of severe mitochondrial cardiomyopathy by gene therapy in a mouse model of Friedreich's ataxia.

Authors:  Morgane Perdomini; Brahim Belbellaa; Laurent Monassier; Laurence Reutenauer; Nadia Messaddeq; Nathalie Cartier; Ronald G Crystal; Patrick Aubourg; Hélène Puccio
Journal:  Nat Med       Date:  2014-04-06       Impact factor: 53.440

5.  Novel Nrf2-Inducer Prevents Mitochondrial Defects and Oxidative Stress in Friedreich's Ataxia Models.

Authors:  Rosella Abeti; Annalisa Baccaro; Noemi Esteras; Paola Giunti
Journal:  Front Cell Neurosci       Date:  2018-07-17       Impact factor: 5.505

6.  Insights into the role of oxidative stress in the pathology of Friedreich ataxia using peroxidation resistant polyunsaturated fatty acids.

Authors:  M Grazia Cotticelli; Andrew M Crabbe; Robert B Wilson; Mikhail S Shchepinov
Journal:  Redox Biol       Date:  2013-07-19       Impact factor: 11.799

7.  Activating frataxin expression by repeat-targeted nucleic acids.

Authors:  Liande Li; Masayuki Matsui; David R Corey
Journal:  Nat Commun       Date:  2016-02-04       Impact factor: 14.919

8.  'Mitochondrial energy imbalance and lipid peroxidation cause cell death in Friedreich's ataxia'.

Authors:  R Abeti; M H Parkinson; I P Hargreaves; P R Angelova; C Sandi; M A Pook; P Giunti; A Y Abramov
Journal:  Cell Death Dis       Date:  2016-05-26       Impact factor: 8.469

Review 9.  Chemistry, mechanism and clinical status of antisense oligonucleotides and duplex RNAs.

Authors:  Xiulong Shen; David R Corey
Journal:  Nucleic Acids Res       Date:  2018-02-28       Impact factor: 16.971

10.  Friedreich's ataxia: autosomal recessive disease caused by an intronic GAA triplet repeat expansion.

Authors:  V Campuzano; L Montermini; M D Moltò; L Pianese; M Cossée; F Cavalcanti; E Monros; F Rodius; F Duclos; A Monticelli; F Zara; J Cañizares; H Koutnikova; S I Bidichandani; C Gellera; A Brice; P Trouillas; G De Michele; A Filla; R De Frutos; F Palau; P I Patel; S Di Donato; J L Mandel; S Cocozza; M Koenig; M Pandolfo
Journal:  Science       Date:  1996-03-08       Impact factor: 47.728
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  14 in total

Review 1.  Emerging therapies in Friedreich's Ataxia.

Authors:  Theresa A Zesiewicz; Joshua Hancock; Shaila D Ghanekar; Sheng-Han Kuo; Carlos A Dohse; Joshua Vega
Journal:  Expert Rev Neurother       Date:  2020-09-21       Impact factor: 4.618

2.  The smoothened agonist SAG reduces mitochondrial dysfunction and neurotoxicity of frataxin-deficient astrocytes.

Authors:  Andrés Vicente-Acosta; Alfredo Giménez-Cassina; Javier Díaz-Nido; Frida Loria
Journal:  J Neuroinflammation       Date:  2022-04-12       Impact factor: 8.322

3.  Defining Transcription Regulatory Elements in the Human Frataxin Gene: Implications for Gene Therapy.

Authors:  Jixue Li; Yanjie Li; Jun Wang; Trevor J Gonzalez; Aravind Asokan; Jill S Napierala; Marek Napierala
Journal:  Hum Gene Ther       Date:  2020-07-13       Impact factor: 4.793

4.  Targeting NRF2 for the Treatment of Friedreich's Ataxia: A Comparison among Drugs.

Authors:  Sara Petrillo; Jessica D'Amico; Piergiorgio La Rosa; Enrico Silvio Bertini; Fiorella Piemonte
Journal:  Int J Mol Sci       Date:  2019-10-21       Impact factor: 5.923

5.  Molecular and Cellular Substrates for the Friedreich Ataxia. Significance of Contactin Expression and of Antioxidant Administration.

Authors:  Antonella Bizzoca; Martina Caracciolo; Patrizia Corsi; Thea Magrone; Emilio Jirillo; Gianfranco Gennarini
Journal:  Molecules       Date:  2020-09-07       Impact factor: 4.411

6.  Mitochondrial damage and senescence phenotype of cells derived from a novel frataxin G127V point mutation mouse model of Friedreich's ataxia.

Authors:  Daniel Fil; Balu K Chacko; Robbie Conley; Xiaosen Ouyang; Jianhua Zhang; Victor M Darley-Usmar; Aamir R Zuberi; Cathleen M Lutz; Marek Napierala; Jill S Napierala
Journal:  Dis Model Mech       Date:  2020-07-27       Impact factor: 5.758

Review 7.  Future Prospects of Gene Therapy for Friedreich's Ataxia.

Authors:  Gabriel Ocana-Santero; Javier Díaz-Nido; Saúl Herranz-Martín
Journal:  Int J Mol Sci       Date:  2021-02-11       Impact factor: 5.923

8.  Targeting 3' and 5' untranslated regions with antisense oligonucleotides to stabilize frataxin mRNA and increase protein expression.

Authors:  Yanjie Li; Jixue Li; Jun Wang; David R Lynch; Xiulong Shen; David R Corey; Darshan Parekh; Balkrishen Bhat; Caroline Woo; Jonathan J Cherry; Jill S Napierala; Marek Napierala
Journal:  Nucleic Acids Res       Date:  2021-11-18       Impact factor: 16.971

Review 9.  Hereditary Ataxia: A Focus on Heme Metabolism and Fe-S Cluster Biogenesis.

Authors:  Deborah Chiabrando; Francesca Bertino; Emanuela Tolosano
Journal:  Int J Mol Sci       Date:  2020-05-26       Impact factor: 5.923

10.  Altered Secretome and ROS Production in Olfactory Mucosa Stem Cells Derived from Friedreich's Ataxia Patients.

Authors:  Sara Pérez-Luz; Frida Loria; Yurika Katsu-Jiménez; Daniel Oberdoerfer; Oscar-Li Yang; Filip Lim; José Luis Muñoz-Blanco; Javier Díaz-Nido
Journal:  Int J Mol Sci       Date:  2020-09-11       Impact factor: 5.923
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