Results 131 to 140 of about 117,595 (294)

Biomarkers of Leucine‐Rich Repeat Kinase 2 (LRRK2) and Lysosomal Dysfunction in Progressive Supranuclear Palsy

Movement Disorders, EarlyView.
Abstract Background Common and rare genetic variants in leucine‐rich repeat kinase 2 (LRRK2) have been linked with sporadic and familial Parkinson's disease (PD). Recently, we discovered that common genetic variation near the LRRK2 locus determined survival in progressive supranuclear palsy (PSP).
Louise‐Kristine Nielsen, Joshua L.I. Frost, David P. Vaughan, Raquel Real, Riona Fumi, Marte Theilmann Jensen, Megan Hodgson, Eleanor J. Stafford, Lesley Wu, Olaf Ansorge, Annelies Quaegebeur, Kieren S.J. Allinson, Thomas T. Warner, Zane Jaunmuktane, Anjum Misbahuddin, P. Nigel Leigh, Boyd C.P. Ghosh, Kailash P. Bhatia, Alistair Church, Christopher Kobylecki, Michele T.M. Hu, James B. Rowe, Alan A. Shomo, Danielle L. Graham, Omar S. Mabrouk, Huw R. Morris, Esther M. Sammler, Edwin Jabbari   +27 more
wiley   +1 more source

Meeting review : ESF workshop on "Impact of nucleic acid chemistry on gene function analysis: antisense, aptamers, ribozymes and RNAi" [PDF]

, 2008
The shortage of functional information compared to the abundance of sequence information characterizes today’s situation in functional genomics. For many years the knock-down of a gene’s product has been the most powerful way of analysing its function ...
Engels, Joachim W. (Prof. Dr.), Heermeier, Kathrin, Prydz, Hans, Reinhard-Rupp, Jutta   +3 more
core  

Eplontersen for Hereditary Transthyretin Amyloidosis With Polyneuropathy: An Exploratory Analysis of Treatment Effect in Male and Female Patients

Muscle &Nerve, EarlyView.
This exploratory analysis of data from the NEURO‐TTRansform trial evaluated sex‐specific responses in patients with ATTRv‐PN who had received eplontersen in NEURO‐TTRansform versus a historical placebo. Eplontersen can lead to substantial decreases in percentage serum TTR levels, arrest neuropathy impairment, and improve patient quality of life ...
Márcia Waddington Cruz, John L. Berk, Yeşim Parman, Morie Gertz, Sami Khella, Markus Weiler, T. Jesse Kwoh, Maksym Pola, Barry Reicher, Jonatan Nåtman, Noel R. Dasgupta, Jonas Wixner   +11 more
wiley   +1 more source

Proteomic Characterization of AS1411 Reveals ATP6AP1 as a Mediator of Triple‐Negative Breast Cancer Progression

PROTEOMICS, EarlyView.
ABSTRACT Triple‐negative breast cancer (TNBC) is an aggressive subtype of breast cancer characterized by high metastatic potential, poor prognosis, and limited effective therapeutic options. In this study, we investigated the molecular mechanisms underlying the anticancer effects of the nucleolin (NCL)‐targeting DNA aptamer AS1411 using label‐free ...
Ha‐Song Bae, Yun‐Jeong Seo, Eun‐Bi Kim, Hyoung‐Min Park, I‐Hsuan Liu, J. Eugene Lee, Jae‐Young Kim   +6 more
wiley   +1 more source

Gene‐specific double‐stranded RNAs induce mortality in the South African mealybug Delottococcus aberiae

Pest Management Science, EarlyView.
Gene‐specific double‐stranded RNAs effectively silence essential genes in the invasive citrus pest Delottococcus aberiae, leading to significant mortality through both microinjection and oral delivery. These results provide proof of concept for RNA interference‐based biopesticides as selective and sustainable tools for citrus integrated pest management.
Carolina Gallego‐Giraldo, Alberto Urbaneja, Omar Ruíz‐Rivero, Verónica Aragonés, Marie Guijarro‐Guijarro, José‐Antonio Daròs, Meritxell Perez‐Hedo   +6 more
wiley   +1 more source

Extrahepatic Gene Editing In Vivo Using Organic Solvent‐Free Lipid Nanoparticles

Small, EarlyView.
This article highlights a novel, organic solvent‐free lipid nanoparticle (LNP) formulation that avoids cholesterol, thereby reducing hepatic accumulation and immune activation. These BLNPs enable potent, flexible, and scalable gene delivery, supporting personalized medicine and diverse nucleic acid applications.
Michael Streiber, Na Liu, Laurianne Simon, Franziska Adermann, Vivien Bachmann, Lucas Gath, Stephanie Hoeppener, Stephanie Schubert, Oliver Werz, Vincent Lapinte, Marie Morille, Michael Bauer, Adrian T. Press, Ulrich S. Schubert, Anja Traeger   +14 more
wiley   +1 more source

Nanopore Electroporation: A New Delivery Method Within the Field of Epigenetic Editing

Small, EarlyView.
Nanopore‐mediated electroporation was used to deliver a CRISPRi system to clonal β‐cells. This enabled targeted downregulation of insulin expression while maintaining high cell viability. ABSTRACT Epigenetic modifications influence gene expression and contribute to type 2 diabetes (T2D), but establishing causality requires targeted modulation of ...
Frida Ekstrand, Sabrina Ruhrmann, Karl Bacos, Sabine Bartel, Pytrick Jellema, Marianne G. Rots, Charlotte Ling, Christelle N. Prinz   +7 more
wiley   +1 more source

The role of voltage-gated calcium channel α2δ-1 in the occurrence and development in myofascial orofacial pain

BMC Oral Health
Patients who suffer from myofascial orofacial pain could affect their quality of life deeply. The pathogenesis of pain is still unclear. Our objective was to assess Whether Voltage-gated calcium channel α2δ-1(Cavα2δ-1) is related to myofascial orofacial ...
Yang Lu, Jingfu Wang, Li Li, Xiaodong Zhang   +3 more
doaj   +1 more source

Potential targets for renal cell carcinoma therapy: Mechanistic research and prospects of CAFs in renal cell carcinoma

VIEW, EarlyView.
Cancer‐associated fibroblasts exert intricate mechanisms in the progression and metastasis of renal cell carcinoma. Existing studies have validated the relevant mechanisms of three signaling pathways, which hold promising potential as novel therapeutic targets.
Ruiqiang Sun, Jiankun Zhang, Meixi Liu, Xiyue Dong, Yuanshan Cui   +4 more
wiley   +1 more source

Ultrasmall nanoparticles for co-delivery of antisense oligonucleotides targeting miR-21 and miR-210 to treat glioblastoma

Journal of Nanobiotechnology
Glioblastoma (GBM) is the most common and aggressive type of brain tumour, with less than 5% of patients surviving more than 5 years. Despite decades of research to understand the underlying pathophysiological causes, it has witnessed very slow progress ...
Ravi Raj Singh, Ritu Kulshreshtha, Amirali Popat   +2 more
doaj   +1 more source