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Advancing AAV technology: From capsid design to scalable manufacturing.

作者信息

Stone Daniel, Mietzsch Mario, Ronzitti Giuseppe

机构信息

Vaccine and Infectious Disease Division, Fred Hutchinson Cancer Center, Seattle, WA, USA.

Department of Biochemistry and Molecular Biology, University of Florida, Gainesville, FL, USA.

出版信息

Mol Ther Methods Clin Dev. 2025 May 7;33(2):101477. doi: 10.1016/j.omtm.2025.101477. eCollection 2025 Jun 12.

Abstract
摘要

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本文引用的文献

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AAV capsids target muscle-resident cells with different efficiencies-A comparative study between AAV8, AAVMYO, and AAVMYO2.
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2
A two-pass anion-exchange chromatography strategy for enrichment of full capsids in manufacturing of adeno-associated viral vectors.
Mol Ther Methods Clin Dev. 2025 Mar 3;33(2):101441. doi: 10.1016/j.omtm.2025.101441. eCollection 2025 Jun 12.
3
AAV vector development, back to the future.
Mol Ther. 2025 May 7;33(5):1903-1936. doi: 10.1016/j.ymthe.2025.03.064. Epub 2025 Apr 3.
4
Advances in AAV capsid engineering: Integrating rational design, directed evolution and machine learning.
Mol Ther. 2025 May 7;33(5):1937-1945. doi: 10.1016/j.ymthe.2025.03.056. Epub 2025 Apr 1.
5
Recombinant AAV batch profiling by nanopore sequencing elucidates product-related DNA impurities and vector genome length distribution.
Mol Ther Methods Clin Dev. 2025 Jan 22;33(1):101417. doi: 10.1016/j.omtm.2025.101417. eCollection 2025 Mar 13.
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Identification of the role of SNARE proteins in rAAV vector production through interaction with the viral MAAP.
Mol Ther Methods Clin Dev. 2024 Dec 5;33(1):101392. doi: 10.1016/j.omtm.2024.101392. eCollection 2025 Mar 13.
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αFAP-specific nanobodies mediate a highly precise retargeting of modified AAV2 capsids thereby enabling specific transduction of tumor tissues.
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Quantitative proteomic analysis of residual host cell protein retention across adeno-associated virus affinity chromatography.
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Structural characterization and epitope mapping of the AAVX affinity purification ligand.
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Predictive power of deleterious single amino acid changes to infer on AAV2 and AAV2-13 capsids fitness.
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