An AAV8 Mutant with Better Transduction in Murine Muscle and Nasal Airway Than AAV8
Abstract
Introduction: Recombinant Adeno-Associated Vectors (AAV) represent nowadays the most largely used platform for in vivo gene therapy. Unfortunately, despite promising results in preclinical and clinical studies, pre-existing immunity against the viral capsid remains one of the major hurdles to the safety and efficiency of AAV-based gene transfer in Humans. Particularly, the impact of pre-existing anti-AAV CD8+ T lymphocytes on gene transfer remains poorly defined. This gap in knowledge can, in part, be attributed to the scarcity of AAV-specific CD8+ T cells among the peripheral blood mononuclear cells (PBMCs), and to the lack of relevant animal models. Experimental approach: In order to set up a more sensitive and comprehensive method to detect and characterise AAV-specific CD8+ T cells among PBMCs, we used Tetramer-Associated Magnetic Enrichment (TAME) to analyse the frequency and phenotype of AAV-specific CD8+ T cells by flow cytometry. To this end, we previously generated AAV-loaded tetramers using the UV-mediated peptide exchange technology.
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BibTeX
@article{vandamme2026aav8,
title = {{An AAV8 Mutant with Better Transduction in Murine Muscle and Nasal Airway Than AAV8}},
author = {Celine Vandamme and Marie Devaux and Nicolas Jaulin and Mickaël Guilbaud and Johanne Le Duff and Leslie Hesnard and Hanane Djamai and Marc Bonneville and Philippe Moullier and Xavier Saulquin and Oumeya Adjali},
journal = {Molecular Therapy},
year = {2026},
month = sep,
doi = {10.1016/s1525-0016(16)33505-5},
url = {https://nantes-universite.hal.science/hal-05742551v1},
note = {CC BY-NC-ND 4.0},
}
RIS
TY - JOUR
TI - An AAV8 Mutant with Better Transduction in Murine Muscle and Nasal Airway Than AAV8
AU - Celine Vandamme
AU - Marie Devaux
AU - Nicolas Jaulin
AU - Mickaël Guilbaud
AU - Johanne Le Duff
AU - Leslie Hesnard
AU - Hanane Djamai
AU - Marc Bonneville
AU - Philippe Moullier
AU - Xavier Saulquin
AU - Oumeya Adjali
PY - 2026
DA - 2026/09/08
JO - Molecular Therapy
DO - 10.1016/s1525-0016(16)33505-5
UR - https://nantes-universite.hal.science/hal-05742551v1
AB - Introduction: Recombinant Adeno-Associated Vectors (AAV) represent nowadays the most largely used platform for in vivo gene therapy. Unfortunately, despite promising results in preclinical and clinical studies, pre-existing immunity against the viral capsid remains one of the major hurdles to the safety and efficiency of AAV-based gene transfer in Humans. Particularly, the impact of pre-existing anti-AAV CD8+ T lymphocytes on gene transfer remains poorly defined. This gap in knowledge can, in part, be attributed to the scarcity of AAV-specific CD8+ T cells among the peripheral blood mononuclear cells (PBMCs), and to the lack of relevant animal models. Experimental approach: In order to set up a more sensitive and comprehensive method to detect and characterise AAV-specific CD8+ T cells among PBMCs, we used Tetramer-Associated Magnetic Enrichment (TAME) to analyse the frequency and phenotype of AAV-specific CD8+ T cells by flow cytometry. To this end, we previously generated AAV-loaded tetramers using the UV-mediated peptide exchange technology.
SN - 1525-0016
SN - 1525-0024
ER -