Results 221 to 230 of about 9,649,206 (293)

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

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

Production of adeno-associated virus vectors for in vitro and in vivo applications. [PDF]

Sci Rep, 2019
Kimura T, Ferran B, Tsukahara Y, Shang Q, Desai S, Fedoce A, Pimentel DR, Luptak I, Adachi T, Ido Y, Matsui R, Bachschmid MM.   +11 more
europepmc   +1 more source

Enhancing Lentiviral Purification: Combining CIM QA Monolithic Columns With Design of Experiments Approach

ELECTROPHORESIS, EarlyView.
Lentiviral vector purification using QA CIM monolith anion‐exchange chromatography was optimized via 96‐well and DOE, increasing recovery from 20% to 90%. The process achieved 3.3‐log host cell protein reduction, decreased particle impurities, and showed high binding capacity, supporting large‐scale manufacturing.
Luka Bevc, Ana Pavšič Rijavec, Valentina Novak, Sandra Potušek Šuleski, Ivana Petrović Koshmak, Maja Leskovec, Hana Jug, Aleš Štrancar   +7 more
wiley   +1 more source

Adeno‐associated virus‐mediated restoration of NaV1.1 in targeted brain regions ameliorates epilepsy in symptomatic Dravet syndrome mice

Epilepsia, EarlyView.
Abstract Objective Dravet syndrome (DS) is a developmental and epileptic encephalopathy primarily caused by haploinsufficiency of the SCN1A gene, which encodes the alpha subunit of NaV1.1 voltage‐gated sodium channel. The disease manifests with febrile and spontaneous seizures, developmental delay, cognitive impairment, and increased risk of sudden ...
Martina Mainardi, Simone Brusco, Serena Gea Giannelli, Vania Broccoli, Gaia Colasante   +4 more
wiley   +1 more source

Chimeric Capsid Proteins Impact Transduction Efficiency of Haploid Adeno-Associated Virus Vectors. [PDF]

Viruses, 2019
Chai Z, Zhang X, Dobbins AL, Frost EA, Samulski RJ, Li C.   +5 more
europepmc   +1 more source

Inherited metabolic epilepsies–established diseases, new approaches

Epilepsia Open, EarlyView.
Abstract Inherited metabolic epilepsies (IMEs) represent the inherited metabolic disorders (IMDs) in which epilepsy is a prevailing component, often determining other neurodevelopmental outcomes associated with the disorder. The different metabolic pathways affected by individual IMEs are the basis of their rarity and heterogeneity.
Itay Tokatly Latzer, Phillip L. Pearl
wiley   +1 more source

Precision therapies for genetic epilepsies in 2025: Promises and pitfalls

Epilepsia Open, EarlyView.
Abstract By targeting the underlying etiology, precision therapies offer an exciting paradigm shift to improve the stagnant outcomes of drug‐resistant epilepsies, including developmental and epileptic encephalopathies. Unlike conventional antiseizure medications (ASMs) which only treat the symptoms (seizures) but have no effect on the underlying ...
Shuyu Wang, Emilio Perucca, Samuel F. Berkovic, Piero Perucca   +3 more
wiley   +1 more source

Genetic developmental and epileptic encephalopathies in adulthood: Gaps in evidence and care

Epilepsia Open, EarlyView.
Abstract Developmental and epileptic encephalopathies (DEEs) are a heterogeneous group of the most severe epilepsies, characterized by seizure onset and neurodevelopmental delay in infancy or childhood, often with additional neurological and systemic comorbidities.
Alex M. Dudley, Peter Widdess‐Walsh, Christine Linehan, Danielle M. Andrade, Norman Delanty, Patrick B. Moloney   +5 more
wiley   +1 more source

Illuminating point‐of‐care diagnostics with aggregation‐induced emission biosensors

FlexMat, EarlyView.
This review highlights the application of aggregation‐induced emission luminogens (AIEgens) in point‐of‐care (POC) diagnostics for disease biomarker detection. It systematically examines a range of biosensing platforms, including lateral flow immunoassays, microfluidic chips, and electrochemiluminescence sensors, alongside their utility in detecting ...
Danni Hu, Yubing Hu, Dong Wang, Ben Zhong Tang   +3 more
wiley   +1 more source