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Opportunities for mitochondrial disease gene therapy
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Nature Reviews Drug Discovery 22, 429-430 (2023)
doi: https://doi.org/10.1038/d41573-023-00067-z
Acknowledgements
P.F.C. is a Wellcome Principal Research Fellow (212219/Z/18/Z), and a UK NIHR Senior Investigator, who receives support from a Wellcome Collaborative Award (224486/Z/21/Z), the Medical Research Council (MRC) Mitochondrial Biology Unit (MC_UU_00028/7), the MRC International Centre for Genomic Medicine in Neuromuscular Disease (MR/S005021/1), the Leverhulme Trust (RPG-2018-408), an MRC research grant (MR/S035699/1) and an Alzheimer's Society Project Grant (AS-PG-18b-022). C.V. is supported by the Telethon Foundation (GGP20013), Associazione Luigi Comini Onlus (MitoFight2) and the Department of Biomedical Sciences—UNIPD (SID2022- VISC_BIRD2222_01. J.v.d.A. is a Wellcome Clinical Research Career Development Fellow (219615/Z/19/Z), who receives support from the Evelyn Trust (21-25), the MRC Mitochondrial Biology Unit (MC_UU_00028/8) and the BBSRC (BB/X00256X/1). This research was supported by the NIHR Cambridge Biomedical Research Centre (BRC-1215-20014). The views expressed are those of the authors and not necessarily those of the NIHR or the Department of Health and Social Care.
References
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Schon, K. R. et al. Use of whole genome sequencing to determine genetic basis of suspected mitochondrial disorders: cohort study. BMJ 375, e066288 (2021).
Falabella, M., Minczuk. M., Hanna, M. G., Viscomi, C. & Pitceathly. R. D. S. Gene therapy for primary mitochondrial diseases: experimental advances and clinical challenges. Nat. Rev. Neurol. 18, 689–698 (2022).
Viscomi, C. & Zeviani, M. Strategies for fighting mitochondrial diseases. J. Intern. Med. 287, 665–684 (2020).
Sabatini, M. T., Xia, T. & Chalmers, M. Pricing and market access challenges in the era of one-time administration cell and gene therapies. Pharmaceut. Med. 36, 265–274 (2022).
Supplementary Information
Competing Interests
The authors declare no competing interests.