Bovine AAV is position as a promising lung-tropic and immune-distinct vector for pulmonary gene therapy, with particular relevance for applications requiring systemic delivery in the presence of pre-existing immunity to conventional serotypes.
This study generated a novel recombinant AAV vector rAAV.hu.hu.S17, derived from the human spleen isolate AAV.hu.S17, and systematically evaluated its capsid features, in vitro transduction, and in vivo tissue tropism.
Wenyan Guo, Jiawen Sun, Fei Wang et al.· Journal of Genetic Engineeri...· 0 citations
It is shown that systemic AAV administration provides potent delivery and uniform transduction of cardiac tissue, outperforming localized injection techniques and establishing an effective AAV approach for transgene transfer into the mouse heart and promote the development of gene therapy for cardiac disorders.
I. Galkin, V. Skopenkova, Maria Y. Shubina et al.· BioTech· 0 citations
The results showed the potential of a combinatorial AAV library for model validation and revealed the human microliver platform-PEG as a reliable system for the development of AAV therapeutics.
Carmen Unzu, Amanda X. Chen, Liliana Mancio-Silva et al.· bioRxiv· 0 citations
The findings indicate sustained gene transfer and transduction for at least 180 days following a single AAV1 administration to the primate lung, with minimal immunopathology, which supports the potential feasibility of extended dosing intervals and informs the development of repeatable AAV-based gene therapies for CF.
Prajita Paul, Tanvi Sharma, M. Afshani et al.· Human Gene Therapy· 0 citations
In supernatant AAV vector recovery assays, AAVR knockout increased supernatant vector genome recovery for selected serotypes in a representative clone, although this effect varied among independently isolated clones.
This work establishes metagenomic mining as a powerful tool for accelerating AAV capsid discovery, identifying isolates with favorable tissue tropisms and resistance to broadly neutralizing antibodies.
E. Lyashenko, Tess Torregrosa, Alexandra B. Ysasi et al.· Journal of Virology· 0 citations
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