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03.11.2018 | Short Communication

FOXRED1 silencing in mice: a possible animal model for Leigh syndrome

verfasst von: Mohamed Salama, Sara El-Desouky, Aziza Alsayed, Mahmoud El-Hussiny, Abdelrahman Moustafa, Yasmeen Taalab, Wael Mohamed

Erschienen in: Metabolic Brain Disease | Ausgabe 1/2019

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Abstract

Leigh syndrome (LS) is one of the most puzzling mitochondrial disorders, which is also known as subacute necrotizing encephalopathy. It has an incidence of 1 in 77,000 live births worldwide with poor prognosis. Currently, there is a poor understanding of the underlying pathophysiological mechanisms of the disease without any available effective treatment. Hence, the inevitability for developing suitable animal and cellular models needed for the development of successful new therapeutic modalities. In this short report, we blocked FOXRED1 gene with small interfering RNA (siRNA) using C57bl/6 mice. Results showed neurobehavioral changes in the injected mice along with parallel degeneration in corpus striatum and sparing of the substantia nigra similar to what happen in Leigh syndrome cases. FOXRED1 blockage could serve as a new animal model for Leigh syndrome due to defective CI, which echoes damage to corpus striatum and affection of the central dopaminergic system in this disease. Further preclinical studies are required to validate this model.

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Titel: FOXRED1 silencing in mice: a possible animal model for Leigh syndrome
verfasst von: Mohamed Salama
Sara El-Desouky
Aziza Alsayed
Mahmoud El-Hussiny
Abdelrahman Moustafa
Yasmeen Taalab
Wael Mohamed
Publikationsdatum: 03.11.2018
Verlag: Springer US
Erschienen in: Metabolic Brain Disease / Ausgabe 1/2019
Print ISSN: 0885-7490
Elektronische ISSN: 1573-7365
DOI: https://doi.org/10.1007/s11011-018-0334-z

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