Results 161 to 170 of about 8,933,868 (252)

Adeno-Associated virus-based approaches for mitochondrial diseases: advances and challenges. [PDF]

Mol Psychiatry
Corrà S, Balmaceda V, Viscomi C.
europepmc   +1 more source

The GPX3‐VCAM1 Axis Gates Pro‐Fibrotic Tubule Cell Fate in Hyperuricemic Nephropathy

Advanced Science, EarlyView.
Hyperuricemia induces NLRP3 inflammasome signaling activation and downregulation of GPX3 in proximal tubular epithelial cells, leading to oxidative stress–driven VCAM1 upregulation, emergence of pro‐fibrotic proximal tubule cells, macrophage recruitment, and renal fibrosis.
Yunfei Qi, Qiang Zhao, Yue Lve, Yaming Yuan, Tingting Zhou, Yue Yuan, Dexi Wu, Yifan Tang, Yan Liu, Zhigang Li, Chunping Jiang, Qiurong Ding   +11 more
wiley   +1 more source

An Expanded Toolbox for Versatile Chemical Editing of Adeno-Associated Virus. [PDF]

Angew Chem Int Ed Engl
Pham Q, Glicksman J, Han B, Koo D, Loynd C, Singha Roy SJ, Chatterjee A.   +6 more
europepmc   +1 more source

CRISPR and Gene Augmentation Rescue Trabecular Meshwork Dysfunction in iPSC Models of Lowe Syndrome

Advanced Science, EarlyView.
By modeling Lowe syndrome using patient‐derived iPSCs, this study establishes a human disease model that faithfully recapitulates OCRL deficiency‐associated ciliary and cytoskeletal defects. The model enables evaluation of both mutation‐agnostic DNA augmentation and CRISPR‐mediated mutation correction strategies, both of which restore OCRL function and
Siyu Chen, Zhiquan Liu, Wenmin Wang, Qing Wang, Tia J. Kowal, Fan Zhang, Grzegorz Walkiewicz, Faruk Hossen, Herbert M. Lachman, Jinqiong Zhou, Yiting Wang, Yang Sun   +11 more
wiley   +1 more source

Anion-exchange chromatography separates structurally heterogeneous and low-potency particles in adeno-associated virus manufacture. [PDF]

Mol Ther Adv
Tsunaka Y, Makihira H, Kono H, Yang Z, Lyu X, Soth S, Fukuhara M, Shibuya R, Yamaguchi Y, Uchiyama S.   +9 more
europepmc   +1 more source

Palmitoyl Acyltransferase Zdhhc17 Promotes Functional Recovery After Spinal Cord Injury by Targeting the Nuclear Transport Factors Kpna2 and Ipo9

Advanced Science, EarlyView.
In this study, we demonstrate that Zdhhc17, acting as a PAT, suppresses neuronal apoptosis and promotes axon regeneration after injury, thereby enhancing functional recovery following SCI. In this context, Kpna2 and Ipo9 serve as novel palmitoylation substrates of Zdhhc17, whose palmitoylation prevents the injury‐induced degradation of their proteins ...
Meixuan Chen, Huan Chen, Sining Ding, Fan Liu, Xiaofei Zheng, Yuan Wang, Ruitu Tian, Huan Li, Peilin Liu, Le Hu, Bin Liu, Limin Rong, Mangmang Li   +12 more
wiley   +1 more source

Comparative evaluation of adeno-associated virus and lentivirus mediated gene transfer in adult rat optic nerve. [PDF]

Exp Eye Res
Kinane C, Koilkonda R, Gomez J, Panchal M, Khuu T, Talla V, Park KK.   +6 more
europepmc   +1 more source

Adaptive Network Stabilization Despite Profound Structural Reorganization in Human Organotypic Cultures

Advanced Science, EarlyView.
Adult human organotypic cortical slices undergo marked neuronal loss, laminar disruption, and glial activation, yet their network activity progressively stabilizes after a variable period. Electrophysiology, longitudinal calcium imaging, and quantitative histology show that structurally remodeled human circuits show adaptive capacity resulting in ...
Estilla Zsófia Tóth, Rebeka Stelcz, Réka Bod, Ábel Petik, Eszter Juhász, Kinga Tóth, Liza Szalai, Nour Essam, Fanni Somogyi, Beatrix Kovács, Amirmahdi Mojtahedzadeh, Attila György Bagó, Loránd Erőss, Péter Orbay, Johanna Petra Szabó, Dániel Fabó, Boglárka Hajnal, Bence Rácz, Daniel Hillier, István Ulbert, Lucia Wittner   +20 more
wiley   +1 more source

Temporal Trajectories of the Tau Aggregate Interactome Reveal Stage‐Specific Vulnerabilities in Alzheimer's Disease

Advanced Science, EarlyView.
Tau populations with different aggregation states associate with distinct sets of proteins in the Alzheimer's disease brain. Proteomic discovery combined with single‐molecule analysis in neurons reveals dynamic associations involving proteostasis, metabolism and RNA biology.
Dorothea Böken, Paula Beltran Lobo, Yunzhao Wu, Lyla A. Rowe, Emre Fertan, Cara L. Croft, Maria Jimenez‐Sanchez, Dezerae Cox, David Klenerman   +8 more
wiley   +1 more source

Adeno-associated virus-induced neurotoxicity is prevented by CpG depletion. [PDF]

Mol Ther Adv
Dunn CE, Carvajal BV, Ma J, Castañeda-Hernandez G, Nguyen AV, Jung C, Suriano CM, Boulanger LM.   +7 more
europepmc   +1 more source