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Design of AAV Vectors for Delivery of RNAi
2019Adeno-associated viral vectors have emerged as an important tool for human gene therapy, having demonstrated high transduction efficiency in a broad range of target tissues, a good safety profile in animal models and human clinical trials, and prospective long-lasting gene expression.
Florie, Borel, Christian, Mueller
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New Recombinant Serotypes of AAV Vectors
Current Gene Therapy, 2005AAV based vectors can achieve stable gene transfer with minimal vector related toxicities. AAV serotype 2 (AAV2) is the first AAV that was vectored for gene transfer applications. However, the restricted tissue tropism of AAV and its low transduction efficiency have limited its further development as vector.
Guangping, Gao +2 more
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AAV Vector Delivery to Cells in Culture
2004Adeno-associated virus (AAV) gene delivery vectors are being investigated as vehicles for gene therapy for a wide variety of hereditary and acquired human diseases. AAV's inability to self-propagate, ability to be maintained as an episome in the transduced cell, and relatively innocuous effects on the immune system make it the vector of choice for ...
Andrew, Smith +2 more
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Design and Construction of Functional AAV Vectors
2011Using the basic principles of molecular biology and laboratory techniques presented in this chapter, researchers should be able to create a wide variety of AAV vectors for both clinical and basic research applications. Basic vector design concepts are covered for both protein coding gene expression and small non-coding RNA gene expression cassettes ...
John T, Gray, Serge, Zolotukhin
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Modification and Labeling of AAV Vector Particles
2011Adeno-associated virus (AAV) has become a versatile vector platform. In recent years, powerful -techniques for the generation of tropism-modified vectors (rAAV-targeting vectors) and for investigation of virus-cell interaction were developed. The following chapter describes strategies for insertion of peptide ligands into the viral capsid and the ...
Hildegard, Büning +3 more
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AAV vectors for cancer gene therapy
Current cancer therapies, including cytotoxic agents, molecularly targeted drugs, and immune-based treatments, are often constrained by therapeutic resistance, systemic toxicity, and inconsistent clinical efficacy. The adeno-associated virus (AAV) vector-based therapy emerges as a promising strategy to address these limitations, owing to its favorable ...Myungeun, Lee +3 more
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[Integration of AAV vectors and insertional mutagenesis].
Medecine sciences : M/S, 2016Recombinant AAV vectors (rAAV) are considered as very efficient tools for in vivo gene transfer. Accordingly, several preclinical and clinical gene therapy trials use these vectors to treat inherited and acquired diseases. rAAV vectors possess the capacity to persist for a long term in the transduced tissue in a transcriptionally active, extra ...
Rossi, Axel, Salvetti, Anna
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AAV Vectors: The Rubik’s Cube of Human Gene Therapy
Molecular Therapy, 2022Richard Jude Samulski, Achille FRANCOIS
exaly
Tracing the fate of AAV vectors in the body
Nature Biotechnology, 2023Nerea Zabaleta, Irene Gil-Farina
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