Results 301 to 310 of about 131,610 (332)
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Cell, 2021
Directed evolution of AAV capsids has been a successful strategy for generating bespoke serotypes to target gene therapies more specifically to the intended tissue. This has now been achieved for the largest organ, skeletal muscle, by selecting for an RGD containing integrin binding heptamer in a hypervariable region of the capsid of AAV9.
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Directed evolution of AAV capsids has been a successful strategy for generating bespoke serotypes to target gene therapies more specifically to the intended tissue. This has now been achieved for the largest organ, skeletal muscle, by selecting for an RGD containing integrin binding heptamer in a hypervariable region of the capsid of AAV9.
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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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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
Douglas M, McCarty +2 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, 2009
Abstract AAV-1 is one of the most promising vectors for gene delivery to skeletal muscle. In this issue of Blood, Mingozzi and colleagues now demonstrate that AAV-1–mediated gene transfer into human skeletal muscle results in activation of capsid-specific T cells ...
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Abstract AAV-1 is one of the most promising vectors for gene delivery to skeletal muscle. In this issue of Blood, Mingozzi and colleagues now demonstrate that AAV-1–mediated gene transfer into human skeletal muscle results in activation of capsid-specific T cells ...
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2011
The precise alteration of sequences by homologous recombination is an important strategy for gene therapies as well as investigating gene function and cellular DNA repair pathways. Inefficient delivery of template DNA to the nucleus using transfection or electroporation methods is one limitation of the frequency of homologous recombination in primary ...
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The precise alteration of sequences by homologous recombination is an important strategy for gene therapies as well as investigating gene function and cellular DNA repair pathways. Inefficient delivery of template DNA to the nucleus using transfection or electroporation methods is one limitation of the frequency of homologous recombination in primary ...
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