Results 141 to 150 of about 245,728 (306)

Calcium Shock Enables Efficient and Programmable Particle Delivery for Genome Editing Applications

open access: yesAdvanced Science, EarlyView.
Classical transfection and transduction are inefficient, particularly with confluent cells and organoids, and lack cell type‐specific programmability. This study presents calcium shock (CaSh), a method that dramatically improves particle delivery into single cells, colonies, and organoids.
Nicole Vo   +12 more
wiley   +1 more source

High-Throughput Screening to Identify Novel Compounds Affecting the Genome Editing Efficiency of CRISPR System

open access: yesMolecules
Genome editing is a promising therapeutic strategy for genetic disorders by modifying the genome precisely, especially the CRISPR/Cas9 system. However, a major limitation of CRISPR/Cas9 in gene therapy is the biosafety issues caused by off-target effects.
Jiasong Chang   +9 more
doaj   +1 more source

Gαi1/3 Is a Novel Regulatory Target for RANKL Signal Transduction and Osteoporosis

open access: yesAdvanced Science, EarlyView.
ABSTRACT Osteoporosis, characterized by progressive bone loss and increased fracture risk, is a growing concern as the population ages. Current treatments, though advanced, remain limited, underscoring the necessity for novel therapeutic targets. Recent studies have shown that the immune system plays a key role in osteoporosis, with osteoclasts driving
Chaowen Bai   +15 more
wiley   +1 more source

Pathogenic Role of FGFR3 Autoantibodies in Small Fiber Neuropathy

open access: yesAdvanced Science, EarlyView.
Autoantibodies against fibroblast growth factor receptor 3 (FGFR3) are identified as pathogenic drivers of pain in small fiber neuropathy. By binding to sensory neurons in dorsal root ganglia, FGFR3 autoantibodies activate MAPK signaling and induce hyperexcitability and mechanical hypersensitivity, establishing FGFR3 autoantibodies as a therapeutic ...
Lyuba Y. Salih   +12 more
wiley   +1 more source

Inhibition of SLC11A1‐Mediated Lysosomal Iron Accumulation in Microglia Promotes Repair Following White Matter Stroke

open access: yesAdvanced Science, EarlyView.
Genetic and pharmacological inhibition of SLC11A1 functioning as an H+/Fe2+ antiporter–mediated lysosomal iron accumulation in microglia promotes lysosomal lumen acidification, increases CTSD expression, enhances lysosomal myelin debris uptake and degradation, and promotes repair following white matter stroke. ABSTRACT White matter stroke (WMS) results
Lingling Qiu   +11 more
wiley   +1 more source

Mapping genetic interactions in cancer: a road to rational combination therapies. [PDF]

open access: yes, 2019
The discovery of synthetic lethal interactions between poly (ADP-ribose) polymerase (PARP) inhibitors and BRCA genes, which are involved in homologous recombination, led to the approval of PARP inhibition as a monotherapy for patients with BRCA1/2 ...
Krogan, Nevan J, Tutuncuoglu, Beril
core  

Versatile CRISPR‐Cas Tools for Gene Regulation in Zebrafish via an Enhanced Q Binary System

open access: yesAdvanced Science, EarlyView.
This study introduces CRISPR‐Q, a transgenic CRISPR‐Cas system leveraging the QFvpr/QUAS binary expression platform in zebrafish. CRISPR‐Q overcomes previous challenges in achieving stable and efficient gene regulation. By enabling precise spatiotemporal control of transcript knockdown (CRISPR‐QKD) and gene activation (CRISPR‐Qa), it provides a ...
Miaoyuan Shi   +13 more
wiley   +1 more source

Super‐Enhancer‐Driven SOX4/SMAD3 Mediate Membrane Remodeling by Regulating Phospholipid Metabolism to Accelerate Leukemia Progression

open access: yesAdvanced Science, EarlyView.
Chronic myeloid leukemia blast phase (CML‐BP) poses a severe therapeutic challenge. This study reveals that the super‐enhancer‐driven transcription factors SOX4 and SMAD3 form a cooperative axis critical for disease progression. They co‐activate the oncogenic kinase AXL and promote phospholipid remodeling via LPCAT1 to facilitate its signaling ...
Enzhe Lou   +22 more
wiley   +1 more source

Cleavage‐Resistant CYLD Protects Against Autoimmune Hepatitis

open access: yesAdvanced Science, EarlyView.
Proteolytic cleavage of the deubiquitinase CYLD emerges as a critical driver of autoimmune hepatitis. TNFα‐induced CYLD loss in macrophages amplifies S100A9‐triggered MAPK activation, leading to excessive chemokine production and hepatic inflammation. Pharmacological inhibition of MEK signaling effectively attenuates experimental disease, highlighting ...
Han Liu   +13 more
wiley   +1 more source

Divergent Regulatory Effects of Jasmonic Acid on Tomato Lycopene Biosynthesis Under Light and Dark Conditions

open access: yesAdvanced Science, EarlyView.
This study reveals the mechanism by which jasmonic acid (JA) regulates lycopene synthesis under light and dark conditions. In light, JA activates SlMYC2, which suppresses SlPIF1a and promotes SlPSY1 expression. In darkness, JA induces the acetyltransferase SlNATA1, which acetylates the dark‐accumulated SlPIF1a, thereby repressing SlPSY1 expression ...
Jiayi Xu   +10 more
wiley   +1 more source

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