Evaluation of the loading capacity and patterns of packaged DNA in AAV genomes of different sizes using long-read sequencing

The loading capacity of adeno-associated virus (AAV) vectors is reportedly 4.7–5.0 kb, which limits the size of genes that can be treated with gene therapy. However, the effects of oversized genomes on the integrity of packaged AAV genomes are poorly understood. Herein, nanopore long-read sequencing...

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Main Authors: Mitsuki Kosaka, Ai Fujita Sajiki, Kosuke Fujita, Kazuhisa Yamada, Kenichi Kawano, Kiichi Hirazawa, Tsuyoshi Matsuno, Masahiko Takada, Nobuhiro Shimozawa, Ken-ichi Inoue, Kenya Yuki, Koji M. Nishiguchi
Format: Article
Language:English
Published: Elsevier 2025-06-01
Series:Molecular Therapy: Methods & Clinical Development
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Online Access:http://www.sciencedirect.com/science/article/pii/S2329050125000695
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