A novel class of self-complementary AAV vectors with multiple advantages based on cceAAV lacking mutant ITR

Self-complementary AAV vectors (scAAV) use a mutant inverted terminal repeat (mITR) for efficient packaging of complementary stranded DNA, enabling rapid transgene expression. However, inefficient resolution at the mITR leads to the packaging of monomeric or subgenomic AAV genomes. These noncanonica...

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Autors principals:	Junping Zhang, Dylan A. Frabutt, Matthew Chrzanowski, Ning Li, Lohra M. Miller, Jiahe Tian, Patrick L. Mulcrone, Anh K. Lam, Benjamin E. Draper, Martin F. Jarrold, Roland W. Herzog, Weidong Xiao
Format:	Artigo
Idioma:	Inglês
Publicat:	Elsevier 2024-03-01
Col·lecció:	Molecular Therapy: Methods & Clinical Development
Matèries:	Adeno-associated virus self-complementary AAV vectors covalently closed-end double-stranded AAV transduction genome integrity
Accés en línia:	http://www.sciencedirect.com/science/article/pii/S2329050124000226
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