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Association of the AAV-PRO questionnaire with established outcome measures in AAV
Abstract 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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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
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Chem-AAV: Chemically Modified AAV for Gene Therapy
Chem-AAV: Chemically Modified AAV for Gene Therapy. 20th Annual Meeting of the American-Society-of-Gene-and-Cell-Therapy. (ASGCT)
Mevel, Mathieu +8 more
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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
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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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AAV Gene Transfer to the Heart
2020Recombinant adeno-associated virus (rAAV) has been widely used for gene therapy. AAV-mediated gene transfer leads to durable protein expression in non-proliferating targeted tissues, which enables long-term modulation of gene expression. Here we describe a rAAV production protocol based on PEI-mediated triple transfection of HEK293T cells, followed by ...
Suya, Wang, Yuxuan, Guo, William T, Pu
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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.
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Biodistribution and Shedding of AAV Vectors
2011Determining the AAV vector biodistribution and shedding is central for the safety assessment of proposed early-phase clinical trials. It is especially crucial in the case of AAV vectors since they are injected directly in situ with no possibility of an intermediate ex vivo step, such as in retroviral-mediated approaches.
Caroline, Le Guiner +2 more
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