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Therapy: Gene Therapy
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Total 17 results found since Jan 2013.
Mechanisms and challenges of nanocarriers as non-viral vectors of therapeutic genes for enhanced pulmonary delivery
J Control Release. 2022 Nov 10:S0168-3659(22)00739-8. doi: 10.1016/j.jconrel.2022.10.061. Online ahead of print.ABSTRACTWith the rapid development of biopharmaceuticals and the outbreak of COVID-19, the world has ushered in a frenzy to develop gene therapy. Therefore, therapeutic genes have received enormous attention. However, due to the extreme instability and low intracellular gene expression of naked genes, specific vectors are required. Viral vectors are widely used attributed to their high transfection efficiency. However, due to the safety concerns of viral vectors, nanotechnology-based non-viral vectors have attrac...
Source: Cancer Control - November 13, 2022 Category: Cancer & Oncology Authors: Hezhi Wang Lu Qin Xin Zhang Jian Guan Shirui Mao Source Type: research
The role of lipid components in lipid nanoparticles for vaccines and gene therapy
Adv Drug Deliv Rev. 2022 Jul 1:114416. doi: 10.1016/j.addr.2022.114416. Online ahead of print.ABSTRACTLipid nanoparticles (LNPs) play a key role in mRNA vaccines against COVID-19. In addition, many preclinical and clinical studies, including the siRNA-LNP product, Onpattro®, highlight that LNPs unlock the potential of nucleic acid-based therapies and vaccines. To understand how this 'key' works, we need to learn about the building blocks that constitute LNPs. In this review, we discuss what each lipid component adds to the LNP delivery platform in terms of size, structure, stability, apparent pKa, nucleic acid encapsulati...
Source: Advanced Drug Delivery Reviews - July 5, 2022 Category: Drugs & Pharmacology Authors: Camilla Hald Albertsen Jayesh Kulkarni Dominik Witzigmann Marianne Lind Karsten Petersson Jens B Simonsen Source Type: research
Delivery Strategies for mRNA Vaccines
AbstractThe therapeutic potential for messenger RNA (mRNA) in infectious diseases and cancer was first realized almost three decades ago, but only in 2018 did the first lipid nanoparticle-based small interfering RNA (siRNA) therapy reach the market with the United States Food and Drug Administration (FDA) approval of patisiran (Onpattro ™) for hereditary ATTR amyloidosis. This was largely made possible by major advances in the formulation technology for stabilized lipid-based nanoparticles (LNPs). Design of the cationic ionizable lipids, which are a key component of the LNP formulations, with an acid dissociation constan...
Source: Pharmaceutical Medicine - January 30, 2022 Category: Drugs & Pharmacology Source Type: research
Contactless delivery of plasmid encoding EGFP in vivo by high-intensity pulsed electromagnetic field
In conclusion, feasibility of HI-PEMF was demonstrated for transfection of different tissues (muscle, skin, tumor) and could have great potential in gene therapy and in DNA vaccination.PMID:34058542 | DOI:10.1016/j.bioelechem.2021.107847
Source: Bioelectrochemistry - May 31, 2021 Category: Biochemistry Authors: Matej Kranjc Simona Kranjc Brezar Gregor Ser ša Damijan Miklav čič Source Type: research
Gene Therapy Leaves a Vicious Cycle
Reena Goswami1, Gayatri Subramanian2, Liliya Silayeva1, Isabelle Newkirk1, Deborah Doctor1, Karan Chawla2, Saurabh Chattopadhyay2, Dhyan Chandra3, Nageswararao Chilukuri1 and Venkaiah Betapudi1,4*
1Neuroscience Branch, Research Division, United States Army Medical Research Institute of Chemical Defense, Aberdeen, MD, United States
2Department of Medical Microbiology and Immunology, University of Toledo College of Medicine and Life Sciences, Toledo, OH, United States
3Roswell Park Comprehensive Cancer Center, Buffalo, NY, United States
4Department of Physiology and Biophysics, Case Western Reserve University, Clev...
Source: Frontiers in Oncology - April 23, 2019 Category: Cancer & Oncology Source Type: research
SOCS and Herpesviruses, With Emphasis on Cytomegalovirus Retinitis
Christine I. Alston1,2 and Richard D. Dix1,2*
1Department of Biology, Viral Immunology Center, Georgia State University, Atlanta, GA, United States
2Department of Ophthalmology, Emory University School of Medicine, Atlanta, GA, United States
Suppressor of cytokine signaling (SOCS) proteins provide selective negative feedback to prevent pathogeneses caused by overstimulation of the immune system. Of the eight known SOCS proteins, SOCS1 and SOCS3 are the best studied, and systemic deletion of either gene causes early lethality in mice. Many viruses, including herpesviruses such as herpes simplex virus and cytomega...
Source: Frontiers in Immunology - April 10, 2019 Category: Allergy & Immunology Source Type: research
Efficient delivery of nucleic acid molecules into skin by combined use of microneedle roller and flexible interdigitated electroporation array
Rationale: Delivery of nucleic acid molecules into skin remains a main obstacle for various types of gene therapy or vaccine applications. Here we propose a novel electroporation approach via combined use of a microneedle roller and a flexible interdigitated electroporation array (FIEA) for efficient delivery of DNA and siRNA into mouse skin.Methods: Using micromachining technology, closely spaced gold electrodes were made on a pliable parylene substrate to form a patch-like electroporation array, which enabled close surface contact between the skin and electrodes. Pre-penetration of the skin with a microneedle roller resu...
Source: Theranostics - June 20, 2018 Category: Molecular Biology Authors: Dong Huang, Deyao Zhao, Xiaoxia Wang, Chunhui Li, Tongren Yang, Lili Du, Zewen Wei, Qiang Cheng, Huiqing Cao, Zicai Liang, Yuanyu Huang, Zhihong Li Tags: Research Paper Source Type: research
Inhibition of West Nile virus Replication by bifunctional siRNA targeting the NS2A and NS5 Conserved region.
CONCLUSION: Thus this bifunctional siRNA intervention paves the new way for therapeutic treatment of WNV disease.
PMID: 29874999 [PubMed - as supplied by publisher]
Source: Current Gene Therapy - June 6, 2018 Category: Genetics & Stem Cells Authors: Karothia D, Dash PK, Parida MM, Bhagyawant S, Kumar JS Tags: Curr Gene Ther Source Type: research
Attenuated Salmonella as a Delivery System for siRNA-Based Tumor Therapy
This technology comprises live, attenuated Salmonella strains as a delivery system for small interfering double-stranded RNA (siRNA)-based tumor therapy. The inventors' data provide the first convincing evidence that Salmonella can be used for delivering plasmid-based siRNAs into tumors growing in vivo. Claimed in the related patent application are methods of inhibiting the growth or reducing the volume of solid cancer tumors using the si-RNA constructs directed against genes that promote tumor survival and cancer cell growth. The Stat3-siRNAs carried by an attenuated S. typhimurium described in the application exhibit tum...
Source: NIH OTT Licensing Opportunities - January 9, 2009 Category: Research Authors: admin Source Type: research
