Gene editing improves endoplasmic reticulum-mitochondrial contacts and unfolded protein response in Friedreich's ataxia iPSC-derived neurons

• Main Authors: Priyanka Mishra (Author), Anusha Sivakumar (Author), Avalon Johnson (Author), Carla Pernaci (Author), Anna S. Warden (Author), Lilas Rony El-Hachem (Author), Emily Hansen (Author), Rafael A. Badell-Grau (Author), Veenita Khare (Author), Gabriela Ramirez (Author), Sydney Gillette (Author), Angelyn B. Solis (Author), Peng Guo (Author), Nicole Coufal (Author), Stephanie Cherqui (Author)
• Format: Book
• Published: Frontiers Media S.A., 2024-02-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Friedreich ataxia (FRDA) is a multisystemic, autosomal recessive disorder caused by homozygous GAA expansion mutation in the first intron of frataxin (FXN) gene. FXN is a mitochondrial protein critical for iron-sulfur cluster biosynthesis and deficiency impairs mitochondrial electron transport chain...