Results 141 to 150 of about 110,545 (302)

Nonviral Gold Nanoparticle‐Mediated Delivery of CRISPR‐Cas9 Ribonucleoprotein and Long DNA Transgenes Into Primary Blood Cells

Advanced NanoBiomed Research, EarlyView.
CRISPR/Cas9 has revolutionized the field of gene therapy, but delivery remains an outstanding issue. We propose a nonviral gold‐nanoparticle platform for co‐delivery of CRISPR/Cas9 ribonucleoprotein and long 2.1 kilobase dsDNA transgene constructs. This CRISPR‐AuNP is inexpensive to produce and mediate gene editing and DNA delivery in T cells and CD34+
Rachel A. Cunningham, Karthikeya S. V. Gottimukkala, Daniel D. Lane, Katrina Poljakov, Patricia Lipson, Mark R. Enstrom, Alessandro Rizzi, Aude G. Chapuis, Jennifer E. Adair   +8 more
wiley   +1 more source

Comparison of Receptor‐Mediated Endocytosis and Its Application to Enhance DNA Transfection by TFAMoplex

Advanced NanoBiomed Research, EarlyView.
We developed an assay to distinguish cellular binding from internalization. Compatible with microscopy and high‐throughput screening, the method identifies ligand‐mediated uptake. Applying top candidates to a protein‐based DNA carrier enhanced transfection efficiency, providing a rational strategy to improve non‐viral gene delivery systems.
David Scherer, Steffen Honrath, Jonas Kurmann, Alexander Klipp, Finn Brigger, Jean‐Christophe Leroux   +5 more
wiley   +1 more source

Photocaged Oxytocin and Vasopressin Probes to Decipher Neuropeptide Signalling With High Spatiotemporal Resolution

Angewandte Chemie International Edition, EarlyView.
Novel and biocompatible photoactivatable oxytocin and vasopressin probes, utilising state‐of‐the‐art photocages, remain inactive in the dark. Light exposure facilitates rapid spatiotemporal receptor activation and phospholipase C (PLC)‐mediated downstream signalling. Figure created in https://BioRender.com.
Konstantin Raabe, Predrag Kalaba, Xuan Ling Hilary Yong, Greta Crudeli, Sarah Melzer, Erik Keimpema, Victor Anggono, Markus Muttenthaler   +7 more
wiley   +1 more source

Improved Detection of Myositis‐Specific Autoantibodies Using Luciferase Immunoprecipitation Systems Assay: Comparison With Line Blot and Conventional Immunoprecipitation

Arthritis &Rheumatology, EarlyView.
Objective Myositis‐specific autoantibodies guide the diagnosis and classification of idiopathic inflammatory myopathies, but current immunoassays vary in accuracy, particularly for autoantibodies associated with interstitial lung disease. To compare the performance of the luciferase immunoprecipitation systems (LIPS) assay with line blot and ...
Peter D. Burbelo, Iago Pinal‐Fernandez, Maria Casal‐Dominguez, Xin Tian, Shijinqiu Gao, Neha Gupta, Eric W. Robbins, John A. Chiorini, Christopher A. Mecoli, Lisa Christopher‐Stine, Livia Casciola‐Rosen, Andrew L. Mammen, Sonye K. Danoff, Anthony F. Suffredini, Julio A. Huapaya   +14 more
wiley   +1 more source

Adeno-associated virus-mediated neuroglobin overexpression ameliorates the N-methyl-N-nitrosourea-induced retinal impairments: a novel therapeutic strategy against photoreceptor degeneration

Therapeutics and Clinical Risk Management, 2017
Ye Tao,1,* Zhen Yang,2,* Wei Fang,2 Zhao Ma,3 Yi Fei Huang,1 Zhengwei Li4 1Department of Ophthalmology, Key Lab of Ophthalmology and Visual Science, Chinese PLA General Hospital, Beijing, 2Department of Neurosurgery, Institute for Functional Brain ...
Tao Y, Yang Z, Fang W, Ma Z, Huang YF, Li Z   +5 more
doaj  

The evolving therapeutic landscape of spinal muscular atrophy – A scoping review of investigational agents, emerging delivery technologies and strategic innovations

British Journal of Clinical Pharmacology, EarlyView.
Spinal muscular atrophy (SMA) is a severe neuromuscular disease with emerging therapeutic complexity. This review aims to systematically map the global pipeline of investigational treatments for SMA. Using ClinicalTrials.gov and complementary international registries, we identified 21 planned or ongoing interventional trials from 2020 to 2025 targeting
Andrej Belančić, Patrick Eustaquio, Elvira Meni Maria Gkrinia, Ivana Stević, Eugen Javor, Yun Wah Lam, Slobodan Janković, Dinko Vitezić   +7 more
wiley   +1 more source

Adaptive Machine Learning Framework for Optimizing the Affinity Purification of Adeno‐Associated Viral Vectors

Biotechnology and Bioengineering, EarlyView.
ABSTRACT Adeno‐associated viral (AAV) vectors for gene therapy are becoming integral to modern medicine, providing therapeutic options for diseases once deemed incurable. Currently, viral vector purification is a critical bottleneck in the gene therapy industry, impacting product efficacy and safety as well as accessibility and cost to patients ...
Kelvin P. Idanwekhai, Shriarjun Shastry, Morgan R. Hurst, Arianna Minzoni, Eduardo Barbieri, Luke Remmler, Eugene N. Muratov, Michael A. Daniele, Stefano Menegatti, Alexander Tropsha   +9 more
wiley   +1 more source

Effectiveness of resveratrol in inducing adeno-associated virus as a potential definitive therapy for SCN5A mutation in Brugada syndrome: a narrative review

The Egyptian Heart Journal
Objectives Brugada syndrome (BrS) is a hereditary channelopathy that affects cardiac electrical signal transmission, with SCN5A gene mutation being the most common cause.
Andin Zahrani Pateda, Andi Alika Azzahra, Kuni Zakiyyah Sumargo, Muchtar Nora Ismail Siregar   +3 more
doaj   +1 more source

A Mechanistic Model of rAAV Production in Synthetic Cell Lines

Biotechnology and Bioengineering, EarlyView.
ABSTRACT The recombinant adeno‐associated virus (rAAV) is a widely used vector for gene therapy. Its manufacturing faces significant challenges in producing the large quantities of vectors needed for clinical applications and reducing empty particles.
Prahalad Srinivasan, Han‐Jung Kuo, Yu‐Chieh Lin, Yen‐An Lu, Wei‐Shou Hu, Qi Zhang   +5 more
wiley   +1 more source

Serotonin 5‐HT7 receptor signaling in neuropsychiatric disorders

Bulletin of the Korean Chemical Society, EarlyView.
5‐HT7R recruits Gs, G12, and β‐arrestin signaling to regulate neuronal plasticity, circuit function, and kinase‐linked intracellular responses. This review summarizes how these pathway‐selective modules contribute to autism spectrum disorder, depression, and schizophrenia, highlighting 5‐HT7R as a pathway‐informed therapeutic target. Abstract Serotonin
Eunseo Park, Hyunah Choo
wiley   +1 more source