title: Mitochondrial DNA disease: new options for prevention.
abstract: 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 mitochondrial 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.
authors.affiliation: Mitochondrial Research Group and Newcastle University Centre for Brain Ageing and Vitality, Institute for Ageing and Health, Newcastle University, Newcastle upon Tyne, UK.
authors.forename: Lyndsey
authors.initials: L
authors.lastname: Craven
authors.forename: Joanna L
authors.initials: JL
authors.lastname: Elson
authors.forename: Laura
authors.initials: L
authors.lastname: Irving
authors.forename: Helen A
authors.initials: HA
authors.lastname: Tuppen
authors.forename: Lisa M
authors.initials: LM
authors.lastname: Lister
authors.forename: Gareth D
authors.initials: GD
authors.lastname: Greggains
authors.forename: Samantha
authors.initials: S
authors.lastname: Byerley
authors.forename: Alison P
authors.initials: AP
authors.lastname: Murdoch
authors.forename: Mary
authors.initials: M
authors.lastname: Herbert
authors.forename: Doug
authors.initials: D
authors.lastname: Turnbull
mesh_terms: D004272:DNA, Mitochondrial / Q000235:genetics*; D005260:Female; D015316:Genetic Therapy / Q000379:methods*; D006801:Humans; D008297:Male; D008928:Mitochondria / Q000235:genetics; D028361:Mitochondrial Diseases / Q000235:genetics / Q000517:prevention & control* / Q000628:therapy*
chemical_list: D004272:DNA, Mitochondrial