Cargando…

Intrathecal delivery of frataxin mRNA encapsulated in lipid nanoparticles to dorsal root ganglia as a potential therapeutic for Friedreich’s ataxia

In Friedreich’s ataxia (FRDA) patients, diminished frataxin (FXN) in sensory neurons is thought to yield the predominant pathology associated with disease. In this study, we demonstrate successful usage of RNA transcript therapy (RTT) as an exogenous human FXN supplementation strategy in vitro and i...

Descripción completa

Detalles Bibliográficos
Autores principales:	Nabhan, Joseph F., Wood, Kristy M., Rao, Varada P., Morin, Jeffrey, Bhamidipaty, Surya, LaBranche, Timothy P., Gooch, Renea L., Bozal, Fazli, Bulawa, Christine E., Guild, Braydon C.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Nature Publishing Group 2016
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4756688/ https://www.ncbi.nlm.nih.gov/pubmed/26883577 http://dx.doi.org/10.1038/srep20019

Ejemplares similares

Perturbation of cellular proteostasis networks identifies pathways that modulate precursor and intermediate but not mature levels of frataxin
por: Nabhan, Joseph F., et al.
Publicado: (2015)

Frataxin levels in peripheral tissue in Friedreich ataxia
por: Lazaropoulos, Michael, et al.
Publicado: (2015)

Posttranslational regulation of mitochondrial frataxin and identification of compounds that increase frataxin levels in Friedreich’s ataxia
por: Hackett, Peter T., et al.
Publicado: (2022)

Frataxin gene editing rescues Friedreich’s ataxia pathology in dorsal root ganglia organoid-derived sensory neurons
por: Mazzara, Pietro Giuseppe, et al.
Publicado: (2020)

Neurodegeneration in Friedreich's Ataxia: From Defective Frataxin to Oxidative Stress
por: Gomes, Cláudio M., et al.
Publicado: (2013)

Selected missense mutations impair frataxin processing in Friedreich ataxia
Publicado: (2017)

Selected missense mutations impair frataxin processing in Friedreich ataxia
por: Clark, Elisia, et al.
Publicado: (2017)

Simultaneous Quantification of Mitochondrial Mature Frataxin and Extra-Mitochondrial Frataxin Isoform E in Friedreich’s Ataxia Blood
por: Wang, Qingqing, et al.
Publicado: (2022)

Iron-dependent regulation of frataxin expression: implications for treatment of Friedreich ataxia
por: Li, Kuanyu, et al.
Publicado: (2008)

Novel Frataxin Isoforms May Contribute to the Pathological Mechanism of Friedreich Ataxia
por: Xia, Haiyan, et al.
Publicado: (2012)

Novel Point Mutations in Frataxin Gene in Iranian Patients with Friedreich’s Ataxia
por: HEIDARI, Mohammad Mehdi, et al.
Publicado: (2014)

DNA methylation in Friedreich ataxia silences expression of frataxin isoform E
por: Rodden, Layne N., et al.
Publicado: (2022)

HDAC Inhibitors Correct Frataxin Deficiency in a Friedreich Ataxia Mouse Model
por: Rai, Myriam, et al.
Publicado: (2008)

Development of Frataxin Gene Expression Measures for the Evaluation of Experimental Treatments in Friedreich’s Ataxia
por: Plasterer, Heather L., et al.
Publicado: (2013)

Extra-mitochondrial mouse frataxin and its implications for mouse models of Friedreich’s ataxia
por: Weng, Liwei, et al.
Publicado: (2020)

Interferon gamma upregulates frataxin and corrects the functional deficits in a Friedreich ataxia model
por: Tomassini, Barbara, et al.
Publicado: (2012)

The Friedreich's ataxia protein frataxin modulates DNA base excision repair in prokaryotes and mammals
por: Thierbach, René, et al.
Publicado: (2010)

Frataxin deficiency increases cyclooxygenase 2 and prostaglandins in cell and animal models of Friedreich's ataxia
por: Hayashi, Genki, et al.
Publicado: (2014)

Dyclonine rescues frataxin deficiency in animal models and buccal cells of patients with Friedreich's ataxia
por: Sahdeo, Sunil, et al.
Publicado: (2014)

Frataxin deficiency alters gene expression in Friedreich ataxia derived IPSC‐neurons and cardiomyocytes
por: Angulo, Mariana B., et al.
Publicado: (2022)

Friedreich ataxia: metal dysmetabolism in dorsal root ganglia
por: Koeppen, Arnulf H, et al.
Publicado: (2013)

Repeat-induced epigenetic changes in intron 1 of the frataxin gene and its consequences in Friedreich ataxia
por: Greene, Eriko, et al.
Publicado: (2007)

A Potential New Therapeutic Approach for Friedreich Ataxia: Induction of Frataxin Expression With TALE Proteins
por: Chapdelaine, Pierre, et al.
Publicado: (2013)

Friedreich's Ataxia, Frataxin, PIP5K1B: Echo of a Distant Fracas
por: Bayot, Aurélien, et al.
Publicado: (2013)

Low apolipoprotein A-I levels in Friedreich’s ataxia and in frataxin-deficient cells: Implications for therapy
por: Wang, QingQing, et al.
Publicado: (2018)

Frataxin‐deficient neurons and mice models of Friedreich ataxia are improved by TAT‐MTScs‐FXN treatment
por: Britti, Elena, et al.
Publicado: (2017)

Dimethyl fumarate dosing in humans increases frataxin expression: A potential therapy for Friedreich’s Ataxia
por: Jasoliya, Mittal, et al.
Publicado: (2019)

Role of frataxin protein deficiency and metabolic dysfunction in Friedreich ataxia, an autosomal recessive mitochondrial disease
por: Clark, Elisia, et al.
Publicado: (2018)

Recent Advances in the Elucidation of Frataxin Biochemical Function Open Novel Perspectives for the Treatment of Friedreich’s Ataxia
por: Monfort, Beata, et al.
Publicado: (2022)

Limitations in a frataxin knockdown cell model for Friedreich ataxia in a high-throughput drug screen
por: Calmels, Nadège, et al.
Publicado: (2009)

The First Cellular Models Based on Frataxin Missense Mutations That Reproduce Spontaneously the Defects Associated with Friedreich Ataxia
por: Calmels, Nadège, et al.
Publicado: (2009)

Time-resolved functional analysis of acute impairment of frataxin expression in an inducible cell model of Friedreich ataxia
por: Poburski, Dörte, et al.
Publicado: (2016)

An AAV9 coding for frataxin clearly improved the symptoms and prolonged the life of Friedreich ataxia mouse models
por: Gérard, Catherine, et al.
Publicado: (2014)

Peptide SS-31 upregulates frataxin expression and improves the quality of mitochondria: implications in the treatment of Friedreich ataxia
por: Zhao, Hongting, et al.
Publicado: (2017)

Correlation between frataxin expression and contractility revealed by in vitro Friedreich’s ataxia cardiac tissue models engineered from human pluripotent stem cells
por: Wong, Andy On-Tik, et al.
Publicado: (2019)

Dorsal root ganglia in Friedreich ataxia: satellite cell proliferation and inflammation
por: Koeppen, Arnulf H., et al.
Publicado: (2016)

Mesenchymal Stem Cells Restore Frataxin Expression and Increase Hydrogen Peroxide Scavenging Enzymes in Friedreich Ataxia Fibroblasts
por: Kemp, Kevin, et al.
Publicado: (2011)

Lentivirus-meditated frataxin gene delivery reverses genome instability in Friedreich ataxia patient and mouse model fibroblasts
por: Khonsari, H, et al.
Publicado: (2016)

Adding a temporal dimension to the study of Friedreich's ataxia: the effect of frataxin overexpression in a human cell model
por: Vannocci, Tommaso, et al.
Publicado: (2018)

SINEUP non-coding RNAs rescue defective frataxin expression and activity in a cellular model of Friedreich's Ataxia
por: Bon, Carlotta, et al.
Publicado: (2019)

Cannot write session to /tmp/vufind_sessions/sess_a6cmq65t1etubfdtu538q3cfnt