Results 181 to 190 of about 89,643 (298)

A single liver-sparing myotropic AAV ameliorates muscular dystrophy in humanized mice by base editing. [PDF]

J Clin Invest
Pien YC, Li H, Bulliard G, Liu H, Durbacz MZ, Xu Z, Chai AC, Chemello F, Sanchez-Ortiz E, Zabaleta N, Vandenberghe LH, Liu N, Olson EN.   +12 more
europepmc   +1 more source

Huntingtin Aggregate‐Responsive Autophagy Gene Circuit Mitigates Disease Pathology in R6/2 Mice

Advanced Science, EarlyView.
CD98‐mediated receptor‐mediated transcytosis enables LIP‐CD98 nanocarriers to cross the blood–brain barrier and deliver ARAA to neurons. mHTT aggregates activate the 11G–NarX sensor, initiating Auto‐P and Trans‐P signaling through the VP48–NarL relay.
Jie Zhu, Xi‐xiu Xie, Lei Li, Chen Tian, Hao‐tian Wang, Xiao‐jie Wang, Xiao‐lin Yu, Gui‐feng Zhang, Rui‐tian Liu   +8 more
wiley   +1 more source

Benchmarking the next generation of brain-penetrant AAV capsids. [PDF]

Mol Ther Adv
Broccoli V, Alberti S.
europepmc   +1 more source

ACAT1‐Mediated SP1‐K694 Lactylation Promotes M1 Macrophage Polarization via TREM1 Transcription in COPD

Advanced Science, EarlyView.
Upon cigarette smoke exposure(CSE), LDHA expression is increased in lung macrophages, promoting the conversion of pyruvate to lactate and elevating the lactylation level of SP1 at the K694 site. This, in turn, enhances SP1 enrichment at the TREM1 promoter, increases TREM1 expression, promoted M1 macrophage polarization, and triggers the secretion of ...
Ling Lin, Ping Zhang, Qing Song, Tao Li, Yuqin Zeng, Ping Chen   +5 more
wiley   +1 more source

One-stage assay factor VIII activity reflects AAV-derived factor VIII-enhanced thrombin activation and predicts phenotype. [PDF]

Blood
Sternberg AR, Watson CT, Davidson RJ, Uguen M, Kiialainen A, George LA.   +5 more
europepmc   +1 more source

LAPTM5 Downregulation‐Driven VAMP8 Phosphorylation Impairs Autophagosome‐Lysosome Fusion and Aggravates Septic Acute Lung Injury

Advanced Science, EarlyView.
LAPTM5 functions as a critical molecular scaffold bridging PGAM5 and VAMP to facilitate PGAM5‐mediated dephosphorylation of VAMP8. This cascade promotes autophagosome‐lysosome fusion, restores impaired autophagic flux, and clears damaged mitochondria, thereby mitigating cellular oxidative stress and systemic inflammation.
Lang Jiang, Qilan Li, Hao Liu, Xuehui Gao, Hongyu Nie, Lang Chen, Mei He, Xuefeng Li, Xiangming Fang, Jiqian Xu, Hao Zhang, Jiahong Xia, You Shang   +12 more
wiley   +1 more source

Early‐Life Manganese Overexposure Activates Microglial Phagocytosis through the YY1/TREM2 Axis Leading to Hippocampal Synaptic Remodeling in Mice

Advanced Science, EarlyView.
Early‐life manganese exposure induces behavioral deficits and aberrant hippocampal synaptic remodeling by enhancing microglial phagocytosis in young adult male mice. Mechanistically, manganese promotes Yin Yang 1 (YY1)/triggering receptor expressed on myeloid cells 2 (TREM2) signaling through enhanced HIF1α‐mediated transcription and suppressed SMURF2 ...
Keyu Chen, Bin He, Kuan Liu, Zhuo Ma, Bin Xu   +4 more
wiley   +1 more source

H4K12 Lactylation Regulates NDUFS7 to Drive Microglia Reverse Electron Transport in Spinal Cord Injury

Advanced Science, EarlyView.
Lactate accumulation after SCI promotes H4K12la lactylation, which upregulates NDUFS7, triggering mitochondrial RET and ROS, enforcing pro‐inflammatory microglial polarization. MM@mPTC, a biomimetic nanotherapeutic, degrades LDHA in activated microglia, reducing H4K12la lactylation and blocking RET, thus curbing ROS, fostering a reparative phenotype ...
Chenglong Hong, Zhichen Jiang, Zhouwei Wu, Chenyu Wu, Jiang Liu, Kaijie Guo, Yihui Liang, Shaobo Xu, Yuchen Jin, Yong Xiao, Lei Guo, Hui Xu, Sunren Sheng, Chenggui Wang   +13 more
wiley   +1 more source

Protocol for quantification of AAV-CRISPR-mediated hepatic gene editing in mice. [PDF]

STAR Protoc
Jarrett KE, Baltazar-Nunez R, Borja CA, Calderon PJ, Lai RW, Cheng A, Paudel M, Rubert GE, Suberski A, Traina OK, Cai SY, Srikanth K, Lagor WR, Tarling EJ, de Aguiar Vallim TQ.   +14 more
europepmc   +1 more source

The Cytoplasmic MicroRNA‐Production Complex Regulates MicroRNA Sorting to Extracellular Vesicles During Synaptogenesis

Advanced Science, EarlyView.
The mechanisms of microRNA cargo sorting to extracellular vesicles, important mediators of cell communication, are not fully understood. This study reveals that the microRNA‐production machinery acts as a ‘hub’ for the sorting of microRNAs to extracellular vesicles, a process regulated by dynamic organelle interactions.
Yuzhou Zeng, Selina Luhmann, Aditi Saha, Marta Orlando, Haider Sami, Timo Glatter, Anna Antoniou   +6 more
wiley   +1 more source