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Total 490 results found since Jan 2013.

Clinical pharmacology of siRNA therapeutics: current status and future prospects
Expert Rev Clin Pharmacol. 2022 Oct 17. doi: 10.1080/17512433.2022.2136166. Online ahead of print.ABSTRACTINTRODUCTION: Small interfering RNA (siRNA) has emerged as a powerful tool for post-transcriptional downregulation of multiple genes for various therapies. Naked siRNA molecules are surrounded by several barriers that tackle their optimum delivery to target tissues such as limited cellular uptake, short circulation time, degradation by endonucleases, glomerular filtration, and capturing by the reticuloendothelial system (RES).AREAS COVERED: This review provides insights into studies that investigate various siRNA-based...
Source: Expert Review of Clinical Pharmacology - October 17, 2022 Category: Drugs & Pharmacology Authors: Ahmed Khaled Abosalha Jacqueline Boyajian Waqar Ahmad Paromita Islam Merry Ghebretatios Sabrina Schaly Rahul Thareja Karan Arora Satya Prakash Source Type: research

I.10 Gene therapy and other novel treatment approaches for CMT
There is still no effective drug treatment available for Charcot-Marie-Tooth disease (CMT). Current management relies on rehabilitation therapy, surgery for skeletal deformities, and symptomatic treatment. The challenge is to find disease-modifying therapies. Several approaches, including gene silencing (by means of ASO, siRNA, CRISPR-Cas9 editing) to counteract the PMP22 gene overexpression in the most frequent CMT1A type are under investigation. PXT3003 is the compound in the most advanced phase for CMT1A, as a second phase-III trial is ongoing.
Source: Neuromuscular Disorders - October 1, 2022 Category: Neurology Authors: D. Pareyson Source Type: research

Multifunctional magnetic nanocarriers for delivery of siRNA and shRNA plasmid to mammalian cells: Characterization, adsorption and release behaviors
This study introduces a new MNP functionalization that can be used for the magnetically driven intracellular delivery of nucleic acids.PMID:36162177 | DOI:10.1016/j.colsurfb.2022.112861
Source: Colloids and Surfaces - September 26, 2022 Category: Biotechnology Authors: Chi-Hsien Liu Cheng-Han Lin Yi-Jun Chen Wei-Chi Wu Chun-Chao Wang Source Type: research

Advanced molecular therapies for neurological diseases: focus on stroke, alzheimer's disease, and parkinson's disease
AbstractNeurological diseases (NDs) are one of the leading causes of disability and the second leading cause of death globally. Among these stroke, Alzheimer's disease (AD), and Parkinson's disease (PD) are the most common NDs. A rise in the absolute number of individuals affected with these diseases indicates that the current treatment strategies in management and prevention of these debilitating diseases are not effective sufficiently. Therefore, novel treatment strategies are being explored to cure these diseases by addressing the causative mechanisms at the molecular level. Advanced therapies like gene therapy (gene ed...
Source: Neurological Sciences - September 6, 2022 Category: Neurology Source Type: research

Chondroitin sulfate modified chitosan nanoparticles as an efficient and targeted gene delivery vehicle to chondrocytes
In conclusion, CH-CS nanoparticles can be considered as a candidate for gene therapy purposes in cartilage diseases.PMID:36049252 | DOI:10.1016/j.colsurfb.2022.112786
Source: Colloids and Surfaces - September 1, 2022 Category: Biotechnology Authors: Naghmeh Akbari Moghadam Fatemeh Bagheri Mohamadreza Baghaban Eslaminejad Source Type: research

Cancers, Vol. 14, Pages 4123: Recent Trends in Nanomedicine-Based Strategies to Overcome Multidrug Resistance in Tumors
lin Cancer is the leading cause of economic and health burden worldwide. The commonly used approaches for the treatment of cancer are chemotherapy, radiotherapy, and surgery. Chemotherapy frequently results in undesirable side effects, and cancer cells may develop resistance. Combating drug resistance is a challenging task in cancer treatment. Drug resistance may be intrinsic or acquired and can be due to genetic factors, growth factors, the increased efflux of drugs, DNA repair, and the metabolism of xenobiotics. The strategies used to combat drug resistance include the nanomedicine-based targeted delivery of drugs an...
Source: Cancers - August 26, 2022 Category: Cancer & Oncology Authors: Muhammad Muzamil Khan Vladimir P. Torchilin Tags: Review Source Type: research