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Genetic and reproductive strategies to prevent mitochondrial diseases

(2025) HUMAN REPRODUCTION UPDATE. 31(4). p.269-306
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Abstract
Mitochondrial DNA (mtDNA) diseases pose unique challenges for genetic counselling and require tailored approaches to address recurrence risks and reproductive options. The intricate dynamics of mtDNA segregation and heteroplasmy shift significantly impact the chances of having affected children. In addition to natural pregnancy, oocyte donation, and adoption, IVF-based approaches can reduce the risk of disease transmission. Prenatal diagnosis (PND) and preimplantation genetic testing (PGT) remain the standard methods for women carrying pathogenic mtDNA mutations; nevertheless, they are not suitable for every patient. Germline nuclear transfer (NT) has emerged as a novel therapeutic strategy, while mitochondrial gene editing has increasingly become a promising research area in the field. However, challenges and safety concerns associated with all these techniques remain, highlighting the need for long-term follow-up studies, an improved understanding of disease mechanisms, and personalized approaches to diagnosis and treatment. Given the inherent risks of adverse maternal and child outcomes, careful consideration of the balance between potential benefits and drawbacks is also warranted. This review will provide critical insights, identify knowledge gaps, and underscore the importance of advancing mitochondrial disease research in reproductive health.
Keywords
mitochondrial DNA (mtDNA), mitochondrial disease, preimplantation genetic testing (PGT), prenatal diagnosis (PND), germline nuclear transfer (NT), mitochondrial gene editing, HEREDITARY OPTIC NEUROPATHY, NUCLEAR TRANSFER TECHNIQUES, GERMINAL VESICLE TRANSFER, IN-VITRO FERTILIZATION, CYTOCHROME-C-OXIDASE, STROKE-LIKE SYNDROME, DNA MUTATIONS, PRENATAL-DIAGNOSIS, EMBRYONIC-DEVELOPMENT, POLAR BODY

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Citation

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MLA
Castelluccio, Noemi, et al. “Genetic and Reproductive Strategies to Prevent Mitochondrial Diseases.” HUMAN REPRODUCTION UPDATE, vol. 31, no. 4, 2025, pp. 269–306, doi:10.1093/humupd/dmaf004.
APA
Castelluccio, N., Spath, K., Li, D., De Coo, I. F. M., Butterworth, L., Wells, D., … Heindryckx, B. (2025). Genetic and reproductive strategies to prevent mitochondrial diseases. HUMAN REPRODUCTION UPDATE, 31(4), 269–306. https://doi.org/10.1093/humupd/dmaf004
Chicago author-date
Castelluccio, Noemi, Katharina Spath, Danyang Li, Irenaeus F M De Coo, Lyndsey Butterworth, Dagan Wells, Heidi Mertes, Joanna Poulton, and Björn Heindryckx. 2025. “Genetic and Reproductive Strategies to Prevent Mitochondrial Diseases.” HUMAN REPRODUCTION UPDATE 31 (4): 269–306. https://doi.org/10.1093/humupd/dmaf004.
Chicago author-date (all authors)
Castelluccio, Noemi, Katharina Spath, Danyang Li, Irenaeus F M De Coo, Lyndsey Butterworth, Dagan Wells, Heidi Mertes, Joanna Poulton, and Björn Heindryckx. 2025. “Genetic and Reproductive Strategies to Prevent Mitochondrial Diseases.” HUMAN REPRODUCTION UPDATE 31 (4): 269–306. doi:10.1093/humupd/dmaf004.
Vancouver
1.
Castelluccio N, Spath K, Li D, De Coo IFM, Butterworth L, Wells D, et al. Genetic and reproductive strategies to prevent mitochondrial diseases. HUMAN REPRODUCTION UPDATE. 2025;31(4):269–306.
IEEE
[1]
N. Castelluccio et al., “Genetic and reproductive strategies to prevent mitochondrial diseases,” HUMAN REPRODUCTION UPDATE, vol. 31, no. 4, pp. 269–306, 2025.
@article{01JPHQWRVS7Q4SE1SMS7QW5F37,
  abstract     = {{Mitochondrial DNA (mtDNA) diseases pose unique challenges for genetic counselling and require tailored approaches to address recurrence risks and reproductive options. The intricate dynamics of mtDNA segregation and heteroplasmy shift significantly impact the chances of having affected children. In addition to natural pregnancy, oocyte donation, and adoption, IVF-based approaches can reduce the risk of disease transmission. Prenatal diagnosis (PND) and preimplantation genetic testing (PGT) remain the standard methods for women carrying pathogenic mtDNA mutations; nevertheless, they are not suitable for every patient. Germline nuclear transfer (NT) has emerged as a novel therapeutic strategy, while mitochondrial gene editing has increasingly become a promising research area in the field. However, challenges and safety concerns associated with all these techniques remain, highlighting the need for long-term follow-up studies, an improved understanding of disease mechanisms, and personalized approaches to diagnosis and treatment. Given the inherent risks of adverse maternal and child outcomes, careful consideration of the balance between potential benefits and drawbacks is also warranted. This review will provide critical insights, identify knowledge gaps, and underscore the importance of advancing mitochondrial disease research in reproductive health.}},
  author       = {{Castelluccio, Noemi and Spath, Katharina and Li, Danyang and De Coo, Irenaeus F M and Butterworth, Lyndsey and Wells, Dagan and Mertes, Heidi and Poulton, Joanna and Heindryckx, Björn}},
  issn         = {{1355-4786}},
  journal      = {{HUMAN REPRODUCTION UPDATE}},
  keywords     = {{mitochondrial DNA (mtDNA),mitochondrial disease,preimplantation genetic testing (PGT),prenatal diagnosis (PND),germline nuclear transfer (NT),mitochondrial gene editing,HEREDITARY OPTIC NEUROPATHY,NUCLEAR TRANSFER TECHNIQUES,GERMINAL VESICLE TRANSFER,IN-VITRO FERTILIZATION,CYTOCHROME-C-OXIDASE,STROKE-LIKE SYNDROME,DNA MUTATIONS,PRENATAL-DIAGNOSIS,EMBRYONIC-DEVELOPMENT,POLAR BODY}},
  language     = {{eng}},
  number       = {{4}},
  pages        = {{269--306}},
  title        = {{Genetic and reproductive strategies to prevent mitochondrial diseases}},
  url          = {{http://doi.org/10.1093/humupd/dmaf004}},
  volume       = {{31}},
  year         = {{2025}},
}

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