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Delivery of mtZFNs into Early Mouse Embryos

McCann, Beverly J. and Cox, Andy and Gammage, Payam A. and Stewart, James B. and Zernicka-Goetz, Magdalena and Minczuk, Michal (2018) Delivery of mtZFNs into Early Mouse Embryos. In: Zinc Finger Proteins: Methods and Protocols. Methods in Molecular Biology. No.1867. Humana Press , New York, NY, pp. 215-228. ISBN 9781493987986.

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Mitochondrial diseases often result from mutations in the mitochondrial genome (mtDNA). In most cases, mutant mtDNA coexists with wild-type mtDNA, resulting in heteroplasmy. One potential future approach to treat heteroplasmic mtDNA diseases is the specific elimination of pathogenic mtDNA mutations, lowering the level of mutant mtDNA below pathogenic thresholds. Mitochondrially targeted zinc-finger nucleases (mtZFNs) have been demonstrated to specifically target and introduce double-strand breaks in mutant mtDNA, facilitating substantial shifts in heteroplasmy. One application of mtZFN technology, in the context of heteroplasmic mtDNA disease, is delivery into the heteroplasmic oocyte or early embryo to eliminate mutant mtDNA, preventing transmission of mitochondrial diseases through the germline. Here we describe a protocol for efficient production of mtZFN mRNA in vitro, and delivery of these into 0.5 dpc mouse embryos to elicit shifts of mtDNA heteroplasmy.

Item Type:Book Section
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Zernicka-Goetz, Magdalena0000-0002-7004-2471
Additional Information:© Springer Science+Business Media, LLC, part of Springer Nature 2018. This work was supported by the Medical Research Council, UK (MC_U105697135). We would like to thank Dr. Carlo Viscomi for help and stimulating discussions during the course of this work and the personnel at Phenomics animal care facilities for skillful technical assistance.
Funding AgencyGrant Number
Medical Research Council (UK)MC_U105697135
Subject Keywords:Mitochondrial disease; mtZFN; Germline; In vitro transcription; Micromanipulation
Series Name:Methods in Molecular Biology
Issue or Number:1867
Record Number:CaltechAUTHORS:20190408-161504340
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Usage Policy:No commercial reproduction, distribution, display or performance rights in this work are provided.
ID Code:94573
Deposited By: George Porter
Deposited On:09 Apr 2019 14:46
Last Modified:16 Nov 2021 17:06

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