Results 191 to 200 of about 326,770 (247)

Continuous Capture of Recombinant Adeno‐Associated Virus Particles Using Twin‐Column CaptureSMB

Biotechnology and Bioengineering, EarlyView.
Continuous twin‐column CaptureSMB enables direct capture of secreted rAAV5 from minimally processed perfusion harvest, bypassing lysis, clarification, and concentration steps. Compared to batch capture, it achieves higher yield (+14.3%), greater productivity (+11.4%), and drastically reduced buffer use (−79.2%), while maintaining critical quality ...
Julia M. Müller, Daniela Tobler, Jules Bühler, Damian Hauri, Richard Plieninger, Sven Göbel, Efe Saygili, Yuki Higuchi, Ryosuke Takahashi, Sebastian Vogg, Thomas Müller‐Späth, Thomas K. Villiger   +11 more
wiley   +1 more source

Demonstration of nanofiltration‐based virus safety for AAV manufacturing using model viruses

Biotechnology Progress, EarlyView.
Abstract Virus safety for biopharmaceutical products has been a well‐established concern for several decades. While various virus reduction steps have been identified and implemented for traditional plasma‐derived and monoclonal antibody products, there are various challenges to achieving the same virus safety in emerging gene therapy products.
Brian Buesing, Tomoki Igi, Daniel Strauss   +2 more
wiley   +1 more source

High‐throughput development and scale‐up of recombinant adeno‐associated virus 5 anion‐exchange chromatography for high‐purity capsid separation

Biotechnology Progress, EarlyView.
High‐throughput anion‐exchange chromatography process development enables scalable purification of high‐purity recombinant adeno‐associated virus capsids. Abstract Recombinant adeno‐associated virus (rAAV) production commonly yields a high proportion of empty and partially filled capsids, necessitating downstream purification to enrich full capsids ...
Kevin Vera, Ivan Gajic, Praveen Kusumanchi, Angela Andaluz, Gabrielle Dela Pena, Clifford A. Froelich, Elizabeth Higgins, Jennifer Johnston, Seyed Pouria Motevalian   +8 more
wiley   +1 more source

Cytoskeleton–Membrane Uncoupling in Duchenne Muscular Dystrophy: Implications for Newborn Screening and Early Protection

Cytoskeleton, EarlyView.
ABSTRACT The cytoskeleton of striated muscle integrates force transmission, mechanotransduction, and sarcolemmal stability through coordinated networks of sarcomeres, costameres, and intermediate filaments. Together, these systems establish mechanical continuity between the contractile apparatus, the sarcolemma, and the extracellular matrix.
Houda Cohen, Nora Hosny, Brian R. Thompson, Joseph M. Metzger   +3 more
wiley   +1 more source

Innate Immune Responses to AAV Vectors [PDF]

Frontiers in Microbiology, 2011
Gene replacement therapy by in vivo delivery of adeno-associated virus (AAV) is attractive as a potential treatment for a variety of genetic disorders. However, while AAV has been used successfully in many models, other experiments in clinical trials and in animal models have been hampered by undesired responses from the immune system.
Giridhara R Jayandharan, George Aslanidi, Ashley Martino   +2 more
exaly   +6 more sources

Adeno-Associated Virus (AAV) Vectors in Cancer Gene Therapy [PDF]

Journal of Controlled Release, 2016
Gene delivery vectors based on adeno-associated virus (AAV) have been utilized in a large number of gene therapy clinical trials, which have demonstrated their strong safety profile and increasingly their therapeutic efficacy for treating monogenic ...
David Schaffer, Jorge L Santiago-Ortiz, Jorge L Santiago-Ortiz   +2 more
exaly   +2 more sources

AAV Vector Development, Back to the Future

Molecular Therapy
Adeno-Associated Virus (AAV) has become a pivotal tool in gene therapy, providing a safe and efficient platform for long-term transgene expression. This review presents a comprehensive analysis of AAV’s historical development, from its initial identification as a “contaminant” to its current clinical applications.
Richard Jude Samulski, Liujiang Song, Anna P Tretiakova   +2 more
exaly   +3 more sources

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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, Douglas M McCarty, R Jude Samulski   +2 more
exaly   +3 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, Phillipe, Moullier, Valder R, Arruda   +2 more
openaire   +2 more sources

AAV vectors and tumorigenicity

Nature Biotechnology, 2007
Chromosomal integration of rAAV vectors may induce hepatocellular carcinoma in neonatally treated mice.
openaire   +1 more source