Article

Contaminating manufacturing plasmids and disrupted vector genomes present in liver tissue following adeno-associated virus gene therapy

2025-01-14

Abstract excerpt

Adeno-associated viruses (AAVs) are the most used vectors in gene therapy but can frequently cause liver complications in patients. The mechanisms underlying AAV-related liver toxicity remain poorly understood, posing challenges for effective prevention and intervention. We undertook long and short read metagenomic sequencing of liver tissue from a child with spinal muscular atrophy type 1 experiencing significant...

Topics

Open a Topic to create a Post that cites this publication.

Identifiers and source

Literature Corpus work: d88e1014-4197-5267-a8e8-ac14d49dccfc
DOI: 10.1101/2025.01.13.25320105

Open publication

Related research

Semantic proximity does not establish scientific evidence.

Click a neighbor to travelStep 1 · 12 closest

Contaminating manufacturing plasmids and disrupted vector genomes present in liver tissue following adeno-associated virus gene therapyDOI 10.1101/2025.01.13.25320105Read

Residual DNA impurities in AAV vectors—nature and transcriptionSemantic proximity Read

Strategies to improve safety profile of AAV vectorsSemantic proximity Read

Gene Therapy Leaves a Vicious CycleSemantic proximity Read

Stowaways in the cargo: Contaminating nucleic acids in rAAV preparations for gene therapySemantic proximity Read

Subgenomic satellite particle generation in recombinant AAV vectors results from DNA lesion/breakage and non-homologous end joiningSemantic proximity Read

DNA-Based Nonviral Gene Therapy─Challenging but PromisingSemantic proximity Read

Adeno-associated viruses for gene therapy – clinical implications and liver-related complications, a guide for hepatologistsSemantic proximity Read

Bibliometric analysis of global research trends in adeno-associated virus vector for gene therapy (1991-2022)Semantic proximity Read

Recombinant Adeno-Associated Virus Gene Therapy in Light of Luxturna (and Zolgensma and Glybera): Where Are We, and How Did We Get Here?Semantic proximity Read

Adeno-associated virus infection and its impact in human health: an overviewSemantic proximity Read

Evaluating the state of the science for adeno-associated virus integration: An integrated perspectiveSemantic proximity Read

Development challenges associated with rAAV-based gene therapiesSemantic proximity Read