Article

Amelioration of Leigh syndrome induced by mouse blastocyst complementation with a mutant human mitochondrial ATP synthase 6

2021-10-21

Abstract excerpt

Mutations in mitochondrial genes are primary causes of multiple inherited disorders, especially affecting tissues with high aerobic energy demands such as the eye, brain, heart, and skeletal muscles. Such mitochondrial diseases are untreatable, have poor prognoses, and relevant animal models are few. Here we microinjected into the mouse blastocyst an adeno-associated virus (AAV) that we redirected to mitochondria...

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Literature Corpus work: 28b27598-3000-5b16-b8af-56901a1a26fc
DOI: 10.21203/rs.3.rs-963913/v1

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