Results 191 to 200 of about 74,668 (263)

Adeno-Associated Virus Toxicity in Duchenne Muscular Dystrophy: Mechanisms and Clinical Considerations. [PDF]

Genes (Basel)
Saylam E, D'ambrosio ES, Pesco MT, Gushchina LV.   +3 more
europepmc   +1 more source

Serotype‐Tailored Midstream and Downstream Operations to Advance the Manufacturing of Adeno‐Associated Viral Vectors

Biotechnology and Bioengineering, EarlyView.
ABSTRACT Adeno‐associated viruses (AAVs) are the leading vectors for in vivo gene therapy, fueling a growing demand for scalable and robust biomanufacturing processes. Yet, the impact of cell lysis conditions on the performance of midstream and downstream unit operations across serotypes remains poorly characterized.
Arianna Minzoni, Shriarjun Shastry, Eduardo Barbieri, Morgan R. Hurst, Kelvin Idanwekhai, Yuxuan Wu, Katherine Ernst, Wai‐Keen Chung, Christopher Bombeck, Saad A. Khan, Sushmita Koley, Gabriella Kiss, Oliver Rammo, Alexander Tropsha, Cristiana Boi, Michael A. Daniele, Steven Cramer, Barry Byrne, Stephen Kaminsky, Angie Snell Bennett, Stefano Menegatti   +20 more
wiley   +1 more source

Distinguishing Pseudotransduction and True Transduction Enables Characterization and Bioengineering of Extracellular Vesicle-Adeno-Associated Virus Vectors. [PDF]

J Extracell Vesicles
Boucher JD, Stranford DM, Edelstein HI, Tullman-Ercek D, Kamat NP, Leonard JN.   +5 more
europepmc   +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

The adeno-associated virus Rep proteins target PP4:SMEK1 by preventing substrate recruitment. [PDF]

PLoS Pathog
Vandewinkel B, Torrekens S, Claes Z, Bollen M, Henckaerts E.   +4 more
europepmc   +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

Improvement of Adeno-Associated Virus (AAV)-Based Technologies by Cell-Penetrating Penta-Peptides (CPP5s). [PDF]

Pharmaceutics
Guo CW, Diener A, Matsuyama S.
europepmc   +1 more source

Impact of single‐use bioreactor bag materials on Spodoptera frugiperda cell expansion for recombinant adeno‐associated virus manufacturing

Biotechnology Progress, EarlyView.
Abstract Recombinant adeno‐associated virus production for gene therapy applications commonly relies on the Spodoptera frugiperda 9 (Sf9) baculovirus infection platform. The process encompasses cell expansion from flasks through intermediate‐scale bioreactors to large‐scale production.
Min Tae Park, Seyed Pouria Motevalian
wiley   +1 more source

Gene Therapy Using Recombinant Adeno-Associated Virus for Leber Congenital Amaurosis Induced by RPE65 Mutation. [PDF]

Clin Ophthalmol
Owliaee I, Teimoori A, Shoushtari M, Shojaeian A.   +3 more
europepmc   +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