Results 251 to 260 of about 79,958 (292)

Association of the AAV-PRO questionnaire with established outcome measures in AAV

open access: yesRheumatology, 2023
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
openaire   +5 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
exaly   +3 more sources

Chem-AAV: Chemically Modified AAV for Gene Therapy

open access: yes, 2017
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
core   +4 more sources

AAV as An Immunogen

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
openaire   +2 more sources

Recombinant AAV Production

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
openaire   +2 more sources

Recombinant AAV Purification

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
openaire   +2 more sources

AAV Gene Transfer to the Heart

2020
Recombinant 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
openaire   +2 more sources

CAPitalizing on AAV

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.
openaire   +2 more sources

Biodistribution and Shedding of AAV Vectors

2011
Determining 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
openaire   +2 more sources

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