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
Adeno-associated virus (AAV)-mediated gene transfer remains a promising strategy for cystic fibrosis (CF), but durability of expression and optimal dosing intervals are unresolved challenges. Here, we evaluated long-term gene transfer, transduction, and immunological responses following a single pulmonary administratio...
Prajita Paul, Tanvi Sharma, Masoud 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.
ABSTRACT Gene therapy using adeno-associated virus (AAV) vectors offers promising treatment for genetic disorders, but significant limitations restrict clinical application. Current AAV serotypes exhibit strong liver tropism and require high doses for extra-hepatic targeting, and pre-existing antibodies (NAbs) exclude...
Unknown authors· Journal of Virology· 0 citations
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