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

Friedreich's ataxia (FRDA) is the most common inherited form of ataxia in humans. It is caused by severe downregulation of frataxin (FXN) expression instigated by hyperexpansion of the GAA repeats located in intron 1 of the FXN gene. Despite numerous studies focused on identifying compounds cap...

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Detalhes bibliográficos
Publicado no:Hum Gene Ther
Main Authors: Li, Jixue, Li, Yanjie, Wang, Jun, Gonzalez, Trevor J., Asokan, Aravind, Napierala, Jill S., Napierala, Marek
Formato: Artigo
Idioma:Inglês
Publicado em: Mary Ann Liebert, Inc., publishers 2020
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC7462031/
https://ncbi.nlm.nih.gov/pubmed/32527155
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2020.053
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