Rapamycin nanoparticles mitigate anti-AAV antibody formation in a mouse model of ornithine transcarbamylase deficiency
Antonio Vicidomini, Florence Boisgerault, Giulia Romano, Corrado Guarnaccia, Pauline Vidal, Fanny Collaud, Leandro R. Soria, Giulia de Sabbata, Novella Tedesco, Michela Lisjak, Himanshi Saxena, Luca Camparini, Alessandra Iaconcig, Laura Moretti, Natasa Skoko, Petr Ilyinskii, Nicola Brunetti-Pierri, Giulia Bortolussi, Giuseppe Ronzitti, Takashi Kei Kishimoto, Andrés F. Muro · 2026
Read Rapamycin nanoparticles mitigate anti-AAV antibody formation in a mouse model of ornithine transcarbamylase deficiency by Antonio Vicidomini, Florence Boisgerault, Giulia Romano, Corrado Guarnaccia, Pauline Vidal, Fanny Collaud, Leandro R. Soria, Giulia de Sabbata, Novella Tedesco, Michela Lisjak, Himanshi Saxena, Luca Camparini, Alessandra Iaconcig, Laura Moretti, Natasa Skoko, Petr Ilyinskii, Nicola Brunetti-Pierri, Giulia Bortolussi, Giuseppe Ronzitti, Takashi Kei Kishimoto, Andrés F. Muro (2026) in the Tower of Babel, a free online library of public-domain works preserved in clean, searchable text. No sign-up. The full text is served as a plain-text file at a predictable URL, where text is present: https://antarctic-labs.com/tower-of-babel/PMC13355710.txt.
Description
Antonio Vicidomini and co-authors tested a tolerance-promoting nanoparticle treatment in 2026 in young mice with a urea cycle disorder. Gene therapy for this liver illness works, but the immune system attacks the delivery virus, which blocks repeat dosing, and children lose the benefit as their liver grows. The particles carry rapamycin, a drug that calms a key immune pathway.
Particles alone briefly eased a waste marker in urine. Gene therapy alone restored the missing enzyme and normalized that marker. Given together, the particles stopped antibody formation against the virus in a dose-linked way. The authors say this may one day allow repeat gene dosing in children, after more tuning.
Catalog record
| CREATOR | Antonio Vicidomini, Florence Boisgerault, Giulia Romano, Corrado Guarnaccia, Pauline Vidal, Fanny Collaud, Leandro R. Soria, Giulia de Sabbata, Novella Tedesco, Michela Lisjak, Himanshi Saxena, Luca Camparini, Alessandra Iaconcig, Laura Moretti, Natasa Skoko, Petr Ilyinskii, Nicola Brunetti-Pierri, Giulia Bortolussi, Giuseppe Ronzitti, Takashi Kei Kishimoto, Andrés F. Muro |
|---|---|
| YEAR | 2026 |
| COLLECTION | PAPERS |
| FORMAT | TXT |
| FILE SIZE | 46912 |
| LICENSE | https://creativecommons.org/licenses/by/4.0 |
| RIGHTS | CC BY |
| SOURCE | Europe PMC |