Gene Therapy for Mitochondrial Diseases: Current Status and Future Perspective

• Main Authors: Alessia Di Donfrancesco (Author), Giulia Massaro (Author), Ivano Di Meo (Author), Valeria Tiranti (Author), Emanuela Bottani (Author), Dario Brunetti (Author)
• Format: Book
• Published: MDPI AG, 2022-06-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Mitochondrial diseases (MDs) are a group of severe genetic disorders caused by mutations in the nuclear or mitochondrial genome encoding proteins involved in the oxidative phosphorylation (OXPHOS) system. MDs have a wide range of symptoms, ranging from organ-specific to multisystemic dysfunctions, w...