Article
Assessment of pre-clinical liver models based on their ability to predict the liver-tropism of AAV vectors
2022-09-30
Abstract excerpt
The liver is a prime target for in vivo gene therapies using recombinant adeno-associated viral vectors (rAAV). Multiple clinical trials have been undertaken for this target in the past 15 years, however we are still to see market approval of the first liver-targeted AAV-based gene therapy. Inefficient expression of the therapeutic transgene, vector-induced liver toxicity and capsid, and/or transgene-mediated imm...
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Identifiers and source
- Literature Corpus work
- 4c181d33-3694-542d-8f13-fc6f5bdc51e3
- DOI
- 10.1101/2022.09.28.510021