Recent research on in vivo and ex vivo gene therapy methods in FRDA animal and cell models showcase its promise as a one-time therapy for FRDA. In this review, we provide an overview on the current and emerging prospects of gene therapy for FRDA, with specific focus on advantages of CRISPR/Cas9-mediated gene editing of FXN as a viable option to restore endogenous frataxin expression. We also assess the potential of ex vivo gene editing in hematopoietic stem and progenitor cells as a potential autologous transplantation therapeutic option and discuss its advantages in tackling FRDA-specific safety aspects for clinical translation.
Sunday, June 19, 2022
Advantages and Limitations of Gene Therapy and Gene Editing for Friedreich’s Ataxia
Sivakumar A and Cherqui S (2022); Front. Genome Ed. 4:903139. doi: 10.3389/fgeed.2022.903139
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