Article

Gene replacement therapy provides benefit in an adult mouse model of Leigh syndrome

2020-01-09

Abstract excerpt

Mutations in nuclear-encoded mitochondrial genes are responsible for a broad spectrum of disorders among which Leigh syndrome (LS) is the most common in infancy. No effective therapies are available for this severe disease mainly because of the limited capabilities of the standard adeno-associated viral (AAV) vectors to transduce both peripheral organs and the central nervous system (CNS) when injected systemicall...

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Literature Corpus work: 895eec37-6c66-5034-b87a-f795a9a6a2b1
DOI: 10.1101/2020.01.08.894881

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