Results 201 to 210 of about 74,668 (263)

Rewriting Renal Fate: The Evolving Landscape of Adeno-Associated Virus-Mediated Kidney Gene Therapies. [PDF]

Kidney360
Hickman PG, Asokan A, Wilson MH.
europepmc   +1 more source

Rethinking Delivery Timing in Prenatally Diagnosed Spinal Muscular Atrophy: A Case Report With 2‐Year Follow‐Up

Annals of the Child Neurology Society, EarlyView.
ABSTRACT Objective We describe the clinical course and outcome of a prenatally diagnosed infant with spinal muscular atrophy (SMA) treated immediately after early‐term birth and highlight considerations regarding delivery timing and neonatal management. Methods This male infant had a family history of SMA type 1A.
Emilie Groulx‐Boivin, Olivier Fortin, Simon Thebault, Unain Ansari, Victoria Nixon, Ariane Belzile, Richard Brown, Jarred Garfinkle, Maryam Oskoui   +8 more
wiley   +1 more source

Rapid multiplex liver gene-editing in mice using adeno-associated virus 8 or lipid nanoparticles. [PDF]

Mol Ther Adv
Wu D, Bolt I, Tolenaars DW, Duijst S, In Het Panhuis W, Hofstraat SRJ, Zwolsman R, van der Meel R, Paulusma CC, van de Graaf SFJ.   +9 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

Optimization of Tangential Flow Filtration for High-Yield, Scalable Downstream Processing of Adeno-Associated Virus. [PDF]

Membranes (Basel)
Cardoso S, Bollmann F, Tappe A.
europepmc   +1 more source

Scn1a‐mediated developmental regulation of prefrontal cortex plasticity and cognition

Epilepsia, EarlyView.
Abstract Objective The voltage‐gated sodium channel Nav1.1, encoded by Scn1a, is essential for γ‐aminobutyric acid (GABA)ergic function, and its alteration is associated with neurological disorders such as Dravet syndrome and Alzheimer's disease. We previously demonstrated that local Nav1.1 dysfunction in the medial prefrontal cortex (mPFC) during ...
Maurizio S. Riga, Mercedes Pérez‐Fernández, Yuniesky Andrade‐Talavera, Benito Domínguez‐Velasco, Antonio Rodríguez‐Moreno, Manuel Álvarez‐Dolado   +5 more
wiley   +1 more source

Generation and characterization of a HEK293 cell line optimized for recombinant adeno-associated virus production. [PDF]

Biotechnol Rep (Amst)
Vona M, Bodenmann I, Perrenoud MA, Buchs R, Yasli PK, Nanni L, Daveau R, Félix A, Stolte J, Hov AK, Chollet B, Schuepbach T, Ley D, Guzman E, Fisch I, Mermod N.   +15 more
europepmc   +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

Adeno-Associated Virus (AAV) [PDF]

, 2008
openaire   +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