Results 41 to 50 of about 74,668 (263)

An Epigenetic Fate Converter Based on Nuclei‐Targeting Lipid Nanoparticles Drives Neuronal Programming of Stem Cells for Spinal Cord Repair

open access: yesAdvanced Functional Materials, EarlyView.
Schematic illustration of LNP‐MPG nuclei‐targeting delivery of HMW‐FGF2 promoting histone acetylation to regulate the fate of DPSCs and treat spinal cord injury. LNPs components include pHMW‐FGF2 plasmid, DSPC, Dlin‐MC3‐DMA, cholesterol, and PEG2000, and are modified with MPG to form HMW‐FGF2@LNP‐MPG (HLM). HLM nuclei‐targets DPSCs to deliver HMW‐FGF2,
Heng Zhou   +6 more
wiley   +1 more source

Extracellular vesicles enhance pulmonary transduction of stably associated adeno‐associated virus following intratracheal administration

open access: yesJournal of Extracellular Vesicles, 2023
Adeno‐associated virus (AAV) vector has shown multiple clinical breakthroughs, but its clinical implementation in inhaled gene therapy remains elusive due to difficulty in transducing lung airway cells.
Gijung Kwak   +13 more
doaj   +1 more source

Microfluidic Nano‐Assembly of Red‐Blood‐Cell (RBC) Lipids and Components for Engineering Extracellular Vesicles

open access: yesAdvanced Healthcare Materials, EarlyView.
Engineered red blood cell‐derived extracellular vesicles (eRBCEVs) are synthesized via controlled microfluidic assembly from native RBC lipids, enabling tunable encapsulation of proteins, nucleic acids, nanoparticles, and viral vectors. The platform demonstrates reproducible nanoscale architecture, preserved membrane composition, and functional cargo ...
Chiranth K. Nagaraj   +23 more
wiley   +1 more source

BAP31 Drives Cartilage Calcification through Disruption of Autophagosome–Lysosome Fusion in Osteoarthritis

open access: yesAdvanced Science, EarlyView.
Mechanical stress activates BAP31 in chondrocytes. BAP31 competes with ATG14 for binding to STX17, disrupting the STX17–ATG14 complex required for autophagosome–lysosome fusion. The resultant autophagic flux blockade drives the generation of autophagy‐derived exosomes, which mediate pathological cartilage calcification in OA. Chondrocyte‐targeted BAP31
Zhi‐hua Xu   +13 more
wiley   +1 more source

Adeno-Associated Virus VP1u Exhibits Protease Activity

open access: yesViruses, 2019
Adeno-associated viruses (AAVs) are being developed for gene delivery applications, with more than 100 ongoing clinical trials aimed at the treatment of monogenic diseases.
Justin J. Kurian   +7 more
doaj   +1 more source

Innate Immunocompetent hiPSC‐Derived Neurospheroids Capture Early CNS Responses to rAAV

open access: yesAdvanced Science, EarlyView.
Knowledge of human CNS immune responses to AAV‐based gene therapies remains limited due to the lack of immune‐competent human models. Here, a hiPSC‐derived 3D neuroimmune platform integrating neurospheroids and microglia is established using stirred‐tank bioreactors.
Catarina M. Gomes   +14 more
wiley   +1 more source

Pharmacology of Recombinant Adeno-associated Virus Production

open access: yesMolecular Therapy: Methods & Clinical Development, 2018
Recombinant adeno-associated viral (rAAV) vectors have been used in more than 150 clinical trials with a good safety profile and significant clinical benefit in many genetic diseases.
Magalie Penaud-Budloo   +3 more
doaj   +1 more source

Disrupted TFEB/GDNF–cAMP/ATP Coupling Underlies Astrocytic Dysfunction and Depression in LRRK2 G2019S Parkinson's Mice

open access: yesAdvanced Science, EarlyView.
ABSTRACT The LRRK2 G2019S mutation, a Parkinson's disease–linked variant, has been associated with depression‐like phenotypes, but mechanisms remain unclear. We chart age‐dependent behavioral changes and astrocyte reactivity in G2019S mice and define a pathway connecting LRRK2 to TFEB/GDNF signaling, cellular energetics, and inflammation.
Longping Yao   +5 more
wiley   +1 more source

Pre‐Regenerative Endothelial Cells Empowered by Antler Blood Exosomes Orchestrate Peripheral Nerve Repair

open access: yesAdvanced Science, EarlyView.
The pre‐regenerative vascular niche (PVN) is essential for nerve repair, yet its endothelial blueprint remains unclear. We identified angiogenic ECs (AECs) as the dominant pre‐regenerative subset and found that antler blood–derived exosomes (AB‐EXO) promote repair via IMP3.
Jinsheng Huang   +11 more
wiley   +1 more source

Effects of cellular prion protein on rapid eye movement sleep deprivation-induced spatial memory impairment

open access: yesJournal of Integrative Neuroscience, 2019
The effects of cellular prion protein on rapid eye movement sleep deprivation-induced spatial memory impairment were investigated, and the related mechanisms explored.
Li Hu   +5 more
doaj   +1 more source

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