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Therapy: Gene Therapy

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Total 490 results found since Jan 2013.

PreS/2-21-Guided siRNA Nanoparticles Target to Inhibit Hepatitis B Virus Infection and Replication
A viable therapy is needed to overcome the deadlock of the incurable chronic hepatitis B (CHB). The prolonged existence of covalently closed circular DNA (cccDNA) and integrated HBV DNA in the nucleus of hepatocytes is the root cause of CHB. As a result, it is critical to successfully suppress HBV DNA replication and eliminate cccDNA. RNA interference has been proven in recent research to silence the expression of target genes and thereby decrease HBV replication. However, siRNA is susceptible to be degraded by RNA enzymes in vivo, making it difficult to deliver successfully and lacking of tissue targeting. To exploit the ...
Source: Frontiers in Immunology - April 29, 2022 Category: Allergy & Immunology Source Type: research

Multiscale modeling of the cellular uptake of C6 peptide-siRNA complexes
In this study, multiscaled computational simulations of these peptides were performed in aqueous media, interrogating the relationship between the structure and behaviour. All atom molecular dynamic (MD) simulation results show that all CPPs show stable α-helical amphipathic secondary structures. Furthermore, docking calculations indicate that the C6 peptides can fit into the major groove of the siRNA double-helix, and once filled, could bind randomly along the minor grooves and to other, previously bound peptides. Coarse grained MD simulations were also used to generate free energy profiles for the dimerization of peptid...
Source: Computational Biology and Chemistry - April 24, 2022 Category: Bioinformatics Authors: P W C M Purijjala P V G M Rathnayake B T Kumara B C M Gunathunge R A A P Ranasinghe D N Karunaratne R J K U Ranatunga Source Type: research

Targeting nucleic acid-based therapeutics to tumors: Challenges and strategies for polyplexes
J Control Release. 2022 Apr 15:S0168-3659(22)00207-3. doi: 10.1016/j.jconrel.2022.04.013. Online ahead of print.ABSTRACTThe current medical reality of cancer gene therapy is reflected by more than ten approved products on the global market, including oncolytic and other viral vectors and CAR T-cells as ex vivo gene-modified cell therapeutics. The development of synthetic antitumoral nucleic acid therapeutics has been proceeding at a lower but steady pace, fueled by a plethora of alternative nucleic acid platforms (from various antisense oligonucleotides, siRNA, microRNA, lncRNA, sgRNA, to larger mRNA and DNA) and several c...
Source: Cancer Control - April 18, 2022 Category: Cancer & Oncology Authors: Victoria C Vetter Ernst Wagner Source Type: research

Principles of translational gene therapy for neuromuscular diseases
This article describes the principles of precision gene therapy for neurogenetic diseases using examples of neuromuscular diseases.DISCUSSION: Various strategies of gene therapy have become established and are being tested in preclinical and clinical trials and evaluated as approved forms for long-term efficacy. The aim of every gene therapy is the modification or introduction of the target gene with initiation of a degradation of dysfunctional proteins. Various techniques, such as gene transfer, gene substitution or gene editing in vivo and ex vivo are now usable. For example, a modification of the pre-mRNA using antisens...
Source: Der Nervenarzt - April 6, 2022 Category: Neurology Authors: B Schoser Source Type: research

Effect of nanoparticle-mediated delivery of SFRP4 siRNA for treating Dupuytren disease
Gene Therapy, Published online: 28 March 2022; doi:10.1038/s41434-022-00330-9Effect of nanoparticle-mediated delivery of SFRP4 siRNA for treating Dupuytren disease
Source: Gene Therapy - March 28, 2022 Category: Genetics & Stem Cells Authors: Rujue Jin Weigang Zhu Jiajun Xu Jianhui Gu Aidong Deng Source Type: research

Molecules, Vol. 27, Pages 2082: Inhibition of Metastatic Hepatocarcinoma by Combined Chemotherapy with Silencing VEGF/VEGFR2 Genes through a GalNAc-Modified Integrated Therapeutic System
Jing Hu Hepatocellular carcinoma (HCC) is a highly malignant tumor related to high mortality and is still lacking a satisfactory cure. Tumor metastasis is currently a major challenge of cancer treatment, which is highly related to angiogenesis. The vascular endothelial growth factor (VEGF)/VEGFR signaling pathway is thus becoming an attractive therapeutic target. Moreover, chemotherapy combined with gene therapy shows great synergistic potential in cancer treatment with the promise of nanomaterials. In this work, a formulation containing 5-FU and siRNA against the VEGF/VEGFR signaling pathway into N-acetyl-galactosam...
Source: Molecules - March 24, 2022 Category: Chemistry Authors: Xunan Li Xiang Wang Nian Liu Qiuyu Wang Jing Hu Tags: Article Source Type: research

Overcoming doxorubicin resistance in cancer: siRNA-loaded nanoarchitectures for cancer gene therapy
Life Sci. 2022 Mar 5:120463. doi: 10.1016/j.lfs.2022.120463. Online ahead of print.ABSTRACTGene therapy can be used as a cancer therapy by affecting signaling networks participating in the aggressive behavior of tumors. Small interfering RNA (siRNA) is a genetic tool employed for gene silencing. The siRNA molecules have a length of 21-22 nucleotides, and are synthetic, short non-coding RNAs. The siRNA molecule should be loaded into the RISC complex to carry out its function to degrade mRNA and reduce protein expression. By targeting oncogenic pathways, siRNA can also promote chemosensitivity and reduce resistance. Doxorubi...
Source: Cancer Control - March 8, 2022 Category: Cancer & Oncology Authors: Mahshid Deldar Abad Paskeh Hamidreza Saebfar Mahmood Khaksary Mahabady Sima Orouei Kiavash Hushmandi Maliheh Entezari Mehrdad Hashemi Amir Reza Aref Michael R Hamblin Hui Li Ang Alan Prem Kumar Ali Zarrabi Saeed Samarghandian Source Type: research

RNA interference-based osteoanabolic therapy for osteoporosis by a bone-formation surface targeting delivery system
In this study, we identified casein kinase-2 interacting protein-1 encoding gene (Ckip-1), a negative regulator of bone formation, as an effective target of small interfering RNAs (siRNAs) for improving bone mass. Moreover, an impressive (DSS)6-Liposome (Lipos) nanoparticle system that could target the bone formation surface was synthesized to enhance the delivery of Ckip-1 siRNA to osteogenic lineage cells. The in vitro results confirmed that the (DSS)6-Lipos system could efficaciously improve the intracellular delivery of Ckip-1 siRNA without obvious cell toxicity. The in vivo application of the delivery system showed sp...
Source: Cell Research - March 1, 2022 Category: Cytology Authors: Ye Gao He Xin Bolei Cai Le Wang Qianxin Lv Yan Hou Fuwei Liu Taiqiang Dai Liang Kong Source Type: research