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

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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Bibliographic Details
Main Authors: 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: Article
Language:English
Published: Elsevier 2024-03-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
Adeno-associated virus
self-complementary AAV vectors
covalently closed-end double-stranded AAV
transduction
genome integrity
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050124000226

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http://www.sciencedirect.com/science/article/pii/S2329050124000226

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