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AAV genome modification for efficient AAV production
Heliyon, 2023 The adeno-associated virus (AAV) is one of the most potent vectors in gene therapy. The experimental profile of this vector shows its efficiency and accepted safety, which explains its increased usage by scientists for the research and treatment of a wide range of diseases.
Pavel Volchkov +2 more
exaly +4 more sources
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Current Gene Therapy, 2007
The first in vivo adeno-associated viral vector (AAV) gene transfer experiments were performed in murine models of muscle directed gene transfer. These studies were remarkable for stable expression of a variety of immunogenic transgenes. These findings were translated to other target organs with multiple therapeutic gene products.
Luk H, Vandenberghe, James M, Wilson
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The first in vivo adeno-associated viral vector (AAV) gene transfer experiments were performed in murine models of muscle directed gene transfer. These studies were remarkable for stable expression of a variety of immunogenic transgenes. These findings were translated to other target organs with multiple therapeutic gene products.
Luk H, Vandenberghe, James M, Wilson
openaire +2 more sources
Integration of Adeno-Associated Virus (AAV) and Recombinant AAV Vectors
Annual Review of Genetics, 2004▪ Abstract The driving interest in adeno-associated virus (AAV) has been its potential as a gene delivery vector. The early observation that AAV can establish a latent infection by integrating into the host chromosome has been central to this interest. However, chromosomal integration is a two-edged sword, imparting on one hand the ability to maintain
Samuel M Young +2 more
exaly +3 more sources
Association of the AAV-PRO questionnaire with established outcome measures in AAV
Rheumatology, 2023Abstract Objectives The ANCA-associated vasculitis (AAV) patient-reported outcome (AAV-PRO) questionnaire was developed to capture the impact of AAV and its treatment. We investigated the association of specific AAV-PRO domains with disease activity and extent, damage, depression, health-related ...
Annika Maunz +5 more
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The insect cell-baculovirus expression vector (IC-BEV) platform has enabled small research-scale and large commercial-scale production of recombinant proteins and therapeutic biologics including recombinant adeno-associated virus (rAAV)-based gene delivery vectors.
Pranav R H, Joshi +1 more
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Pranav R H, Joshi +1 more
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Purification of rAAV is a crucial unit operation of the AAV production process. It enables the capture of AAV and removal of contaminants such as host cell proteins, host cell DNA, and other cell culture-related impurities. Here we describe the purification of rAAV produced in insect cells Sf9/rBEV by immuno-affinity capture chromatography.
Pranav R H, Joshi +1 more
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Pranav R H, Joshi +1 more
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Blood, 2006
Comment on Jiang et al, page [107][1] In this issue of Blood, Jiang and colleagues demonstrate long-term therapeutic efficacy in hemophilia A mice and dogs following liver-directed factor VIII gene therapy with AAV vectors.
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Comment on Jiang et al, page [107][1] In this issue of Blood, Jiang and colleagues demonstrate long-term therapeutic efficacy in hemophilia A mice and dogs following liver-directed factor VIII gene therapy with AAV vectors.
openaire +2 more sources
Various AAV Serotypes and Their Applications in Gene Therapy: An Overview
Cells, 2023Alisa Shaimardanova +2 more
exaly