期刊
HUMAN MOLECULAR GENETICS
卷 20, 期 -, 页码 R168-R174出版社
OXFORD UNIV PRESS
DOI: 10.1093/hmg/ddr373
关键词
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资金
- Muscular Dystrophy Campaign
- Wellcome Trust [074454/Z/041Z]
- Medical Research Council [G0601157, G0601943]
- One North East
- UK NIHR Biomedical Research Centre for Ageing and Age-Related Disease
- Newcastle University Centre for Brain Ageing and Vitality
- BBSRC
- EPSRC
- ESRC
- MRC: as part of the Cross-council Lifelong Health and Wellbeing Initiative [G0700718]
- MRC [G90/63, G0601157, G0601943, G0700718] Funding Source: UKRI
- Medical Research Council [G90/63, G0601157, G0601943, G0700718] Funding Source: researchfish
Very recently, two papers have presented intriguing data suggesting that prevention of transmission of human mitochondrial DNA (mtDNA) disease is possible. [Craven, L., Tuppen, H.A., Greggains, G.D., Harbottle, S.J., Murphy, J.L., Cree, L.M., Murdoch, A.P., Chinnery, P.F., Taylor, R.W., Lightowlers, R.N. et al. (2010) Pronuclear transfer in human embryos to prevent transmission of mitochondria! DNA disease. Nature, 465, 82-85. Tachibana, M., Sparman, M., Sritanaudomchai, H., Ma, H., Clepper, L., Woodward, J., Li, Y., Ramsey, C., Kolotushkina, O. and Mitalipov, S. (2009) Mitochondrial gene replacement in primate offspring and embryonic stem cells. Nature, 461, 367-372.] These recent advances raise hopes for families with mtDNA disease; however, the successful translational of these techniques to clinical practice will require further research to test for safety and to maximize efficacy. Furthermore, in the UK, amendment to the current legislation will be required. Here, we discuss the clinical and scientific background, studies we believe are important to establish safety and efficacy of the techniques and some of the potential concerns about the use of these approaches.
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