Selection and evaluation of clinically relevant AAV variants in a xenograft liver model
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DOI: 10.1038/nature12875
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Cited by:
- Adriana Gonzalez-Sandoval & Katja Pekrun & Shinnosuke Tsuji & Feijie Zhang & King L. Hung & Howard Y. Chang & Mark A. Kay, 2023. "The AAV capsid can influence the epigenetic marking of rAAV delivered episomal genomes in a species dependent manner," Nature Communications, Nature, vol. 14(1), pages 1-11, December.
- Fatma-Elzahraa Eid & Albert T. Chen & Ken Y. Chan & Qin Huang & Qingxia Zheng & Isabelle G. Tobey & Simon Pacouret & Pamela P. Brauer & Casey Keyes & Megan Powell & Jencilin Johnston & Binhui Zhao & K, 2024. "Systematic multi-trait AAV capsid engineering for efficient gene delivery," Nature Communications, Nature, vol. 15(1), pages 1-14, December.
- Clément Pontoizeau & Marcelo Simon-Sola & Clovis Gaborit & Vincent Nguyen & Irina Rotaru & Nolan Tual & Pasqualina Colella & Muriel Girard & Maria-Grazia Biferi & Jean-Baptiste Arnoux & Agnès Rötig & , 2022. "Neonatal gene therapy achieves sustained disease rescue of maple syrup urine disease in mice," Nature Communications, Nature, vol. 13(1), pages 1-13, December.
- Mercedes Barzi & Tong Chen & Trevor J. Gonzalez & Francis P. Pankowicz & Seh Hoon Oh & Helen L. Streff & Alan Rosales & Yunhan Ma & Sabrina Collias & Sarah E. Woodfield & Anna Mae Diehl & Sanjeev A. V, 2024. "A humanized mouse model for adeno-associated viral gene therapy," Nature Communications, Nature, vol. 15(1), pages 1-10, December.
- Helena Costa-Verdera & Fanny Collaud & Christopher R. Riling & Pauline Sellier & Jayme M. L. Nordin & G. Michael Preston & Umut Cagin & Julien Fabregue & Simon Barral & Maryse Moya-Nilges & Jacomina K, 2021. "Hepatic expression of GAA results in enhanced enzyme bioavailability in mice and non-human primates," Nature Communications, Nature, vol. 12(1), pages 1-16, December.
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