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Identification of AAV serotypes for lung gene therapy in human embryonic stem cell-derived lung organoids

23 Oct 2020

Abstract excerpt

Gene therapy is being investigated for a range of serious lung diseases, such as cystic fibrosis and emphysema. Recombinant adeno-associated virus (rAAV) is a well-established, safe, viral vector for gene delivery with multiple naturally occurring and artificial serotypes available displaying alternate cell, tissue, and species-specific tropisms. Efficient AAV serotypes for the transduction of the conducting...

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PMID: 33097094
DOI: 10.1186/s13287-020-01950-x

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