Results 171 to 180 of about 32,863 (259)

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

AAV vectors trigger DNA damage response-dependent pro-inflammatory signalling in human iPSC-derived CNS models and mouse brain. [PDF]

Nat Commun
Costa-Verdera H, Meneghini V, Fitzpatrick Z, Abou Alezz M, Fabyanic E, Huang X, Dzhashiashvili Y, Ahiya A, Mangiameli E, Valeri E, Crivicich G, Piccolo S, Cuccovillo I, Caccia R, Chan YK, Bertin B, Ronzitti G, Engel EA, Merelli I, Mingozzi F, Gritti A, Kuranda K, Kajaste-Rudnitski A.   +22 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

Progress report on new epilepsy treatments: A summary of the Eighteenth Eilat Conference on New Antiepileptic Drugs and Devices (EILAT XVIII). II. Treatments in more advanced clinical development

Epilepsia, EarlyView.
Abstract This article summarizes data for 13 investigational treatments for which at least preliminary seizure outcome data in patients with epilepsy were reported at the Eighteenth Eilat Conference on New Antiepileptic Drugs and Devices held in Madrid, Spain, on May 3–6, 2026.
Meir Bialer, Cecilie Johannessen Landmark, Matthias J. Koepp, Emilio Perucca, Piero Perucca, Torbjörn Tomson, H. Steve White, Elaine Wirrell   +7 more
wiley   +1 more source

Identification of SLC35A1 as an essential host factor for the transduction of multi-serotype recombinant adeno-associated virus (AAV) vectors. [PDF]

mBio
Zhang X, Hao S, Feng Z, Ning K, Aksu Kuz C, McFarlin S, Richart D, Cheng F, Zhang-Chen A, McFarlane R, Yan Z, Qiu J.   +11 more
europepmc   +1 more source

Anterior cingulate cortex neuron subtypes differentially regulate seizures

Epilepsia, EarlyView.
Abstract Objective This study aimed to investigate the regulatory roles of distinct neuronal subtypes within the anterior cingulate cortex (ACC) in acute seizures and to identify cell type‐specific mechanisms underlying seizure modulation in this region. Methods Acute seizure models were established in mice via pentylenetetrazol injection.
Ziqian Yan, Ting Tang, Kaishan Wang, Bin Fu, Zesheng Li, Mingshan Liu, Hongyi Huang, Yanfeng Yang, Qiang Zheng, Yangmin Zheng, Yongzhi Shan, Yumin Luo, Guoguang Zhao   +12 more
wiley   +1 more source

Introduction to AAV Vector special issue [PDF]

Gene Therapy, 2008
openaire   +2 more sources

Epilepsy‐associated SCN2A‐L1342P mutation drives network hyperexcitability and widespread transcriptomic changes in human cortical organoids

Epilepsia, EarlyView.
Abstract Objective SCN2A pathogenic mutations, such as the recurrent heterozygous Nav1.2‐L1342P, are monogenic causes of epilepsy. In this human‐induced pluripotent stem cell–derived model system, we aim to investigate the molecular and cellular mechanisms underlying SCN2A‐L1342P‐associated pathology. Methods Using a human male induced pluripotent stem
Maria I. Olivero‐Acosta, Morgan Robinson, Zhefu Que, Zaiyang Zhang, Karen V. Salazar‐Salazar, Hope Elizabeth Harlow, Vinayak Shankar, Seoyeon Hong, Muhan Wang, Conrad M. Otterbacher, Hina Kadono, Manasi Halurkar, Harish Kothandaraman, Nadia Atallah Lanman, Trang Nguyen, Kyle Wettschurack, Benjamin Zirkle, Layan Yunis, Ningren Cui, Xiaoling Chen, Jingliang Zhang, Jiaxiang Wu, William C. Skarnes, Chongli Yuan, Feng Guo, Megan Abbott, Yang Yang   +26 more
wiley   +1 more source

Development of Stable Packaging and Producer Cell Lines for the Production of AAV Vectors. [PDF]

Microorganisms
Merten OW.
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