Novel AAV variants with improved tropism for human Schwann cells
Abstract
Gene therapies and associated technologies are transforming biomedical research and enabling novel therapeutic options for patients living with debilitating and incurable genetic disorders. The vector system based on recombinant adeno-associated viral vectors (AAVs) has shown great promise in recent clinical trials for genetic diseases of multiple organs, such as the liver and the nervous system. Despite recent successes toward the development of novel bioengineered AAV variants for improved transduction of primary human tissues and cells, vectors that can efficiently transduce human Schwann cells (hSCs) have yet to be identified. Here, we report the application of the functional transduction-RNA selection method in primary hSCs for the development of AAV variants for specific and efficient transgene delivery to hSCs. The two identified capsid variants, Pep2hSC1 and Pep2hSC2, show conserved potency for delivery across various in vitro , in vivo , and ex vivo models of hSCs. These novel AAV capsids will serve as valuable research tools, forming the basis for therapeutic solutions for both SC-related disorders or peripheral nervous system injury.
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BibTeX
@article{drouyer2026novel,
title = {{Novel AAV variants with improved tropism for human Schwann cells}},
author = {Matthieu Drouyer and Tak-Ho Chu and Elodie Labit and Florencia Haase and Renina Gale Navarro and Deborah Nazareth and Nicole Rosin and Jessica Merjane and Suzanne Scott and Marti Cabanes-Creus and Adrian Westhaus and Erhua Zhu and Rajiv Midha and Ian Alexander and Jeff Biernaskie and Samantha Ginn and Leszek Lisowski},
journal = {Molecular Therapy - Methods and Clinical Development},
year = {2026},
month = sep,
doi = {10.1016/j.omtm.2024.101234},
url = {https://hal.science/hal-05772028v1},
note = {CC BY},
}
RIS
TY - JOUR
TI - Novel AAV variants with improved tropism for human Schwann cells
AU - Matthieu Drouyer
AU - Tak-Ho Chu
AU - Elodie Labit
AU - Florencia Haase
AU - Renina Gale Navarro
AU - Deborah Nazareth
AU - Nicole Rosin
AU - Jessica Merjane
AU - Suzanne Scott
AU - Marti Cabanes-Creus
AU - Adrian Westhaus
AU - Erhua Zhu
AU - Rajiv Midha
AU - Ian Alexander
AU - Jeff Biernaskie
AU - Samantha Ginn
AU - Leszek Lisowski
PY - 2026
DA - 2026/09/30
JO - Molecular Therapy - Methods and Clinical Development
DO - 10.1016/j.omtm.2024.101234
UR - https://hal.science/hal-05772028v1
AB - Gene therapies and associated technologies are transforming biomedical research and enabling novel therapeutic options for patients living with debilitating and incurable genetic disorders. The vector system based on recombinant adeno-associated viral vectors (AAVs) has shown great promise in recent clinical trials for genetic diseases of multiple organs, such as the liver and the nervous system. Despite recent successes toward the development of novel bioengineered AAV variants for improved transduction of primary human tissues and cells, vectors that can efficiently transduce human Schwann cells (hSCs) have yet to be identified. Here, we report the application of the functional transduction-RNA selection method in primary hSCs for the development of AAV variants for specific and efficient transgene delivery to hSCs. The two identified capsid variants, Pep2hSC1 and Pep2hSC2, show conserved potency for delivery across various in vitro , in vivo , and ex vivo models of hSCs. These novel AAV capsids will serve as valuable research tools, forming the basis for therapeutic solutions for both SC-related disorders or peripheral nervous system injury.
SN - 2329-0501
ER -