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Optimization of adeno-associated viral (AAV) gene therapies vectors for balancing efficacy, longevity and safety for clinical application

2 months 1 week ago
Adeno-associated viral (AAV) vectors are an ideal platform for gene therapy due to their ability to deliver therapeutic cargos safely and effectively across various target organs. Their low immunogenicity contributes to long-lasting therapeutic effects. However, recent insights highlight the significance of CpG content within AAV vectors, where unmethylated CpG dinucleotides can trigger a TLR9-mediated immune response, leading to the rapid elimination of transduced cells. Clinical evidence...
Neel Mehta

In Science Journals

2 months 1 week ago
Science, Volume 387, Issue 6741, Page 1367-1369, March 2025.
Michael Funk, and Melisa Yashinski, Leslie K. Ferrarelli, Marc S. Lavine, Madeleine Seale, Jake S. Yeston, Ekeoma Uzogara, Ian S. Osborne, Di Jiang, Stella M. Hurtley, Yevgeniya Nusinovich, Jesse Smith, Sacha Vignieri, Peter Stern, Corinne N. Simonti,…

In the ashes

2 months 1 week ago
Science, Volume 387, Issue 6741, Page 1343-1346, March 2025.
Warren Cornwall