Results 191 to 200 of about 1,457,553 (300)

Augmenting and Assaying Nav1.1 Protein Quantity for Dravet Syndrome Therapy

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Dravet Syndrome (DS) is a developmental and epileptic encephalopathy predominantly caused by heterozygous loss‐of‐function variants in SCN1A, which encodes Nav1.1. Conserved upstream open reading frames (uORFs) in SCN1A were validated to regulate translation in reporter assays, demonstrating the therapeutic viability of increasing Nav1.1 from ...
Aiswarya Saravanan, Jordan Safran, Anna Loughman, Grace Lin, Shivanshi Vaid, Dana Allababidi, Peter M. Tessier, Louis T. Dang   +7 more
wiley   +1 more source

<i>In vivo</i> delivery strategies for therapeutic CRISPR genome editing. [PDF]

Int J Biol Sci
Martin L, Bohinc J, Recchia A, Gritti S, Santilli G, Zeyland J, Vidaković M, Grdović N, Benabdellah K, Ortiz-Bueno M, Butuner BD, Pisaniello L, Stilhano R, Benati D, Hapil FZ, Khawaja S, Nair RR, Giacomelli C, Atilla E, Zinghirino F, Ferrari T, Corradi F, Laufer TJ, Khnykin D, Aseguinolaza GG, Skrbinek M, Mlakar T, Lapanja T, Lainšček D.   +28 more
europepmc   +1 more source

The Price of Precision: A Critical Review of Molecular Diagnostics in Glioma, From Guidelines to Global Disparities

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Gliomas have undergone a profound redefinition over the past decade, transitioning from morphology‐based entities to biologically coherent diseases defined by molecular alterations. The 2021 WHO Classification of Tumors of the Central Nervous System and its 2022 update formalize this shift, establishing integrated diagnosis as the global ...
Maria Guarnaccia, Sebastiano Cavallaro
wiley   +1 more source

Genome editing precisely boosts salt-tolerant japonica rice yield. [PDF]

J Integr Plant Biol
Zhou M, Sun Q, Sun J, An J, Lv Y, Li C, Lu Y, Qiao J, Zheng X, Qian Q.   +9 more
europepmc   +1 more source

Artificial Intelligence in Systemic Sclerosis: Clinical Applications, Challenges, and Future Directions

Arthritis Care &Research, EarlyView.
Systemic sclerosis (SSc) is a rare autoimmune disease defined by immune dysregulation, vasculopathy, and progressive fibrosis of the skin and internal organs. Despite advances in care, major complications such as interstitial lung disease (ILD) and myocardial involvement remain the leading causes of morbidity and mortality.
Cristiana Sieiro Santos, José Miguel Sequí‐Sabater, Diego Benavent   +2 more
wiley   +1 more source

CRISPR Genome Editing and the Future of Leukaemia Immunotherapy. [PDF]

Health Sci Rep
Rai D, Terranova U.
europepmc   +1 more source

OntOMat: Toward Ontology‐Based Product and Process Design Engineering and Optimization Solutions Fueling Circular Value Chains

Advanced Engineering Materials, EarlyView.
The OntOMat ontology establishes a structured framework for polymer matrix fiber reinforced composite materials, integrating manufacturing processes, characterization methods, and multiscale design through the VDI/VDE 3682 formalized process description standard.
Nicolas Christ, Jörg Hohe, Martin Kördel, Michael Giese, Niklas Toffert, Christian Seidel, Olaf Rehme, Patrik Schneider, Oliver Focke, Elie Abi Aoun, Claas Falldorf, Beñat Gutierrez, Alexandra Kühn, Sven Torstrick-von der Lieth, Christoph Herden, Erik Johannsen, Jan‐Uwe R. Schmidt, Felix Winkelmann, Andreas Schott, Daniel Schott   +19 more
wiley   +1 more source

Affordable CRISPR RNP-Based Genome Editing in Euglena gracilis. [PDF]

Curr Protoc
Minami A, Shimizu M, Tamaki S, Nishinarizki V, Yosua, Mochida K.   +5 more
europepmc   +1 more source

Myogenic Fusogen‐Engineered Lipid Nanoparticles Enhance mRNA Delivery in Skeletal Muscle

Advanced Functional Materials, EarlyView.
This study reports a biomimetic strategy of engineering full‐length Myomaker, a muscle‐specific fusogen, into lipid nanoparticles (LNPs) to harness the native myoblast fusion capability for skeletal muscle mRNA delivery. The resulting Mymk‐LNPs enhance transfection in differentiating myocytes and enable Cre‐mediated reporter activation in injured ...
Fangyu Zhang, Zhisheng Xu, Yaou Duan, Yehui Sun, Shiying Wu, Tao Long, Mateusz Durbacz, Li Zhang, Yufen Xiao, Yu‐Chung Pien, Erick D. Guerrero, Zeru Tian, Xu Wang, Pratima Basak, Sumanta Chatterjee, Ning Liu, Xiaochun Li, Eric N. Olson, Daniel J. Siegwart   +18 more
wiley   +1 more source

Compact 9dBEs Enable Efficient and Precise Genome Editing in Mammalian Cells and In Vivo. [PDF]

Adv Sci (Weinh)
Xiao Q, Tian Z, Weng L, Xu K, Han D, Wu Z, Li J, Li S, Zhang Q, Zhao X, Li G, Wei Y, Gu Z.   +12 more
europepmc   +1 more source