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Nanotechnology-based gene therapy as a credible tool in the treatment of Alzheimer's disease.
Neural Regen Res
October 2023
Institutes for Systems Genetics, Frontiers Science Center for Diseaserelated Molecular Network, West China Hospital, Sichuan University, Chengdu, Sichuan Province, China; King Fahd Medical Research Center, King Abdulaziz University, Jeddah, Saudi Arabia; Department of Pharmacy, Faculty of Allied Health Sciences, Daffodil International University, Dhaka, Bangladesh; Enzymoics, Novel Global Community Educational Foundation, Hebersham, NSW, Australia.
Toxic aggregated amyloid-β accumulation is a key pathogenic event in Alzheimer's disease. Treatment approaches have focused on the suppression, deferral, or dispersion of amyloid-β fibers and plaques. Gene therapy has evolved as a potential therapeutic option for treating Alzheimer's disease, owing to its rapid advancement over the recent decade.
View Article and Find Full Text PDFWhole Exome Sequencing reveals NOTCH1 mutations in anaplastic large cell lymphoma and points to Notch both as a key pathway and a potential therapeutic target.
Haematologica
June 2021
Department of Pathology, University of Cambridge, Cambridge, UK.
Article Synopsis
- * Whole-Exome Sequencing identified mutations enriched in the T-cell receptor and Notch pathways, notably the T349P variant in NOTCH1, which occurs in 12% of ALCL cases and promotes cell growth.
- * Targeting NOTCH1 with γ-secretase inhibitors, especially in combination with the ALK inhibitor Crizotinib, shows potential as an effective treatment strategy, even for Crizotinib-resistant ALCL patients.
A Unique Gene-Silencing Approach, Using an Intelligent RNA Expression Device (iRed), Results in Minimal Immune Stimulation When Given by Local Intrapleural Injection in Malignant Pleural Mesothelioma.
Molecules
April 2020
Department of Pharmacokinetics and Biopharmaceutics, Institute of Biomedical Sciences, Tokushima University, 1-78-1, Sho-machi, Tokushima 770-8505, Japan.
Background: We have recently introduced an intelligent RNA expression device (iRed), comprising the minimum essential components needed to transcribe short hairpin RNA (shRNA) in cells. Use of iRed efficiently produced shRNA molecules after transfection into cells and alleviated the innate immune stimulation following intravenous injection.
Methods: To study the usefulness of iRed for local injection, the engineered iRed encoding luciferase shRNA (Luc iRed), complexed with cationic liposomes (Luc iRed/liposome-complexes), was intrapleurally injected into an orthotopic mesothelioma mouse model.
Metronomic S-1 dosing and thymidylate synthase silencing have synergistic antitumor efficacy in a colorectal cancer xenograft model.
Cancer Lett
August 2017
Department of Pharmacokinetics and Biopharmaceutics, Institute of Biomedical Sciences, Tokushima University, Tokushima, Japan; Department of Cancer and Metabolism, Institute of Biomedical Sciences, Tokushima University, Tokushima, Japan. Electronic address:
Metronomic chemotherapy is currently considered an emerging therapeutic option in clinical oncology. S-1, an oral formulation of Tegafur (TF), a prodrug of 5-fluorouracil (5-FU), is designed to improve the antitumor activity of 5-FU in tandem with reducing its toxicity. Clinically, metronomic S-1 dosing has been approved for the standard first- and second-line treatment of metastatic or advanced stage of colorectal (CRC).
View Article and Find Full Text PDFThe Voltage-dependent Anion Channel 1 Mediates Amyloid β Toxicity and Represents a Potential Target for Alzheimer Disease Therapy.
J Biol Chem
December 2015
From the Department of Life Sciences and the National Institute for Biotechnology in the Negev, Ben-Gurion University of the Negev, Beer Sheva 84105, Israel and
The voltage-dependent anion channel 1 (VDAC1), found in the mitochondrial outer membrane, forms the main interface between mitochondrial and cellular metabolisms, mediates the passage of a variety of molecules across the mitochondrial outer membrane, and is central to mitochondria-mediated apoptosis. VDAC1 is overexpressed in post-mortem brains of Alzheimer disease (AD) patients. The development and progress of AD are associated with mitochondrial dysfunction resulting from the cytotoxic effects of accumulated amyloid β (Aβ).
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