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Advantages and Limitations of Gene Therapy and Gene Editing for Friedreich’s Ataxia

Friedreich’s ataxia (FRDA) is an inherited, multisystemic disorder predominantly caused by GAA hyper expansion in intron 1 of frataxin (FXN) gene. This expansion mutation transcriptionally represses FXN, a mitochondrial protein that is required for iron metabolism and mitochondrial homeostasis, lead...

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Detalles Bibliográficos
Autores principales:	Sivakumar, Anusha, Cherqui, Stephanie
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Frontiers Media S.A. 2022
Materias:	Genome Editing
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9157421/ https://www.ncbi.nlm.nih.gov/pubmed/35663795 http://dx.doi.org/10.3389/fgeed.2022.903139

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