A dual AAV system enables the Cas9-mediated correction of a metabolic liver disease in newborn mice
University of Pennsylvania · Sichuan University · +5 more institutions
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Citation impact
554
total citations
- FWCI
- 41.66
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- 100%
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12Topics & keywords
Topics
Keywords
- Ornithine transcarbamylase
- Ornithine transcarbamylase deficiency
- Urea cycle
- Hyperammonemia
- Reversion
- Biology
- Cas9
- Genetic enhancement
UN Sustainable Development Goals
- Good health and well-being
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