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Session Overview |
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Session 4.3: Therapy 3: reproductive options and mtDNA editing
Invited Speaker: M. Herbert; M. Minczuk
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Presentations | ||
Invited
ID: 2109 Invited Speakers Mitochondrial replacement in action 1Newcastle University, United Kingdom; 2Newcastle Fertility Centre Bibliography
N. Costa-Borges et al., First pilot study of maternal spindle transfer for the treatment of repeated in vitro fertilization failures in couples with idiopathic infertility. Fertil Steril, S0015-0282(23)00136-X (2023). Invited
ID: 2108 Invited Speakers The therapeutic potential of mitochondrial genome engineering MRC Mitochondrial Biology Unit, University of Cambridge, Cambridge, UK Bibliography
Michal Minczuk is a MRC Investigator at the MRC Mitochondrial Biology Unit (MBU) at University of Cambridge, leading a research programme in mitochondrial genetics. His programme encompasses the development of methods for controlled editing of the mammalian mitochondrial genome, mechanistic studies of mitochondrial gene maintenance and expression in health and disease, and the development of advanced gene therapies for mtDNA dysfunction. Oral presentation
ID: 160 Therapy 3: reproductive options and mtDNA editing MitoKO: A library of base editors for the precise ablation of all protein-coding genes in the mouse mitochondrial genome MRC Mitochondrial Biology Unit, University of Cambridge, Cambridge, UK Bibliography
Silva-Pinheiro, P., Mutti, C.D., Van Haute, L. et al. A library of base editors for the precise ablation of all protein-coding genes in the mouse mitochondrial genome. Nat. Biomed. Eng (2022). https://doi.org/10.1038/s41551-022-00968-1 Silva-Pinheiro, P., Nash, P.A., Van Haute, L. et al. In vivo mitochondrial base editing via adeno-associated viral delivery to mouse post-mitotic tissue. Nat Commun 13, 750 (2022). https://doi.org/10.1038/s41467-022-28358-w Silva-Pinheiro, P., Minczuk, M. The potential of mitochondrial genome engineering. Nat Rev Genet 23, 199–214 (2022). https://doi.org/10.1038/s41576-021-00432-x Oral presentation
ID: 174 Therapy 3: reproductive options and mtDNA editing Risk of mtDNA reversal among children born after mitochondrial replacement therapy 1Oregon Health & Science University, United States of America; 2Center for Embryonic Cell and Gene Therapy, Oregon Health and Science University, United States of America Bibliography
First pilot study of maternal spindle transfer for the treatment of repeated in vitro fertilization failures in couples with idiopathic infertility. Fertility and Sterility, 2023 Flash Talk
ID: 155 Therapy 3: reproductive options and mtDNA editing Specific elimination of m.3243A>G mutant mitochondria DNA using mitoARCUS 1Precision BioSciences - Durham, NC, United States of America; 2University of Miami - Miami, FL, United States of America Bibliography
Shoop WK, Gorsuch CL, Bacman SR, Moraes CT. Precise and simultaneous quantification of mitochondrial DNA heteroplasmy and copy number by digital PCR. J Biol Chem. 2022;298(11):102574. doi:10.1016/j.jbc.2022.102574 Flash Talk
ID: 453 Therapy 3: reproductive options and mtDNA editing MitoCRISPR/Cas9 shifts mtDNA heteroplasmy not as effective as other site-specific nucleases. 1Novosibirsk State University, Novosibirsk, Russia; 2Institute of Cytology and Genetics SB RAS, Novosibirsk, Russia; 3Skolkovo Institute of Science and Technology, Moscow, Russia Bibliography
1.Tanaka, M.; Borgeld, H.-J.; Zhang, J.; Muramatsu, S.; Gong, J.-S.; Yoneda, M.; Maruyama, W.; Naoi, M.; Ibi, T.; Sahashi, K.; et al. Gene therapy for mitochondrial disease by delivering restriction endonuclease SmaI into mitochondria. J. Biomed. Sci. 2002, 9, 534–41. https://doi.org/10.1159/000064726. 2. Zakirova, E.G.; Vyatkin, Y.V.; Verechshagina, N.A.; Muzyka, V.V.; Mazunin, I.O.; Orishchenko, K.E. Study of the effect of the introduction of mitochondrial import determinants into the gRNA structure on the activity of the gRNA/SpCas9 complex in vitro.Vavilov Journal of Genetics and Breeding 2020, 24(5):512-518. https://doi.org/10.18699/VJ20.643. 3.Silva-Pinheiro, P., Minczuk, M. The potential of mitochondrial genome engineering. Nat Rev Genet 23, 199–214 (2022). https://doi.org/10.1038/s41576-021-00432-x. 4. Zakirova, E.G.; Muzyka, V.V.; Mazunin, I.O.; Orishchenko, K.E. Natural and Artificial Mechanisms of Mitochondrial Genome Elimination. Life 2021, 11, 76. https://doi.org/10.3390/life11020076. Flash Talk
ID: 271 Therapy 3: reproductive options and mtDNA editing Prenatal diagnostics for a family with 13513G>A mtDNA mutation associated with Leigh Syndrome 1Center for Embryonic Cell and Gene Therapy, Oregon Health and Science University, United States of America; 2Division of Reproductive Endocrinology, Department of Obstetrics and Gynecology, Oregon Health and Science University, United States of America |