Article

Bioengineering novel AAV9-mGULO-GT for multi-disease gene therapy: Targeting mutated GULO expression to cure scurvy and brain diseases.

2023-06-26

Abstract excerpt

<title>Abstract</title> <p>Current clinical breakthroughs in gene therapy have brought adeno-associated virus (AAV) vectors to the forefront of gene delivery systems. Vitamin C deficiency due to GULO mutations is a genetic disorder affecting guinea pigs and humans. In our study, we used AAV9-mGULO-GT to deliver the mouse GULO gene to guinea pigs and restore Vc synthesis in affected tissues, including the liver an...

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Literature Corpus work: b7f01b08-b5e8-5a86-8b7a-bada58210c8d
DOI: 10.21203/rs.3.rs-3028525/v1

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Bioengineering novel AAV9-mGULO-GT for multi-disease gene therapy: Targeting mutated GULO expression to cure scurvy and brain diseases.DOI 10.21203/rs.3.rs-3028525/v1Read

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