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Therapy: Gene Therapy

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Total 490 results found since Jan 2013.

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: Biochemical and Biophysical Research communications - March 1, 2022 Category: Biochemistry Authors: Ye Gao He Xin Bolei Cai Le Wang Qianxin Lv Yan Hou Fuwei Liu Taiqiang Dai Liang Kong Source Type: research

Preparation and Characterization of a Liver Targeted, Poly(amidoamine) Based, Gene Delivery System
Methods Mol Biol. 2022;2455:319-332. doi: 10.1007/978-1-0716-2128-8_24.ABSTRACTNonalcoholic steatohepatitis (NASH) is an aggressive liver disease that is considered a major cause of liver cirrhosis and hepatocellular carcinoma. NASH is characterized by multiple underlying genetic mutations, with no approved cure to date. Gene therapies that target those genetic mutations may play a major role in treating this disease, once delivered specifically to the hepatocytes. In this chapter we present, in detail, the synthesis and the characterization of an efficient gene delivery system capable of targeting hepatocytes by exploitin...
Source: Mol Biol Cell - February 25, 2022 Category: Molecular Biology Authors: Kareem Ebeid Sean M Geary Aliasger K Salem Source Type: research

Synthetic Conjugated Oligoelectrolytes Are Effective siRNA Transfection Carriers: Relevance to Pancreatic Cancer Gene Therapy
Biomacromolecules. 2022 Feb 9. doi: 10.1021/acs.biomac.1c01498. Online ahead of print.ABSTRACTConjugated oligoelectrolyte COE-S6 contains an elongated conjugated core with three cationic charges at each termini of the internal core. As an analogue of bolaamphiphiles, these structural attributes lead to the formation of spherical nanoplexes with Dh = 205 ± 5.0 nm upon mixing with small interfering RNA (siRNA). COE-S6/siRNA nanocomplexes were shown to be protective toward RNase, stimulate endosome escape, and achieve transfection efficiencies comparable to those achieved with commercially available LIP3000. Moreover, COE-S6...
Source: Biomacromolecules - February 9, 2022 Category: Biochemistry Authors: Chengbin Yang Haoqiang Huang Nishtha Manish Singh Cheng Zhou Guang Yang Zhourui Xu Haoming Lin Gaixia Xu Ken-Tye Yong Guillermo C Bazan Source Type: research

Novel Nanotechnological Approaches for Targeting Dorsal Root Ganglion (DRG) in Mitigating Diabetic Neuropathic Pain (DNP)
Diabetic neuropathy is the most entrenched complication of diabetes. Usually, it affects the distal foot and toes, which then gradually approaches the lower part of the legs. Diabetic foot ulcer (DFU) could be one of the worst complications of diabetes mellitus. Long-term diabetes leads to hyperglycemia, which is the utmost contributor to neuropathic pain. Hyperglycemia causing an upregulation of voltage-gated sodium channels in the dorsal root ganglion (DRG) was often observed in models of neuropathic pain. DRG opening frequency increases intracellular sodium ion levels, which further causes increased calcium channel open...
Source: Frontiers in Endocrinology - February 8, 2022 Category: Endocrinology Source Type: research

Gene therapy: Comprehensive overview and therapeutic applications
Life Sci. 2022 Feb 3:120375. doi: 10.1016/j.lfs.2022.120375. Online ahead of print.ABSTRACTGene therapy is the product of man's quest to eliminate diseases. Gene therapy has three facets namely, gene silencing using siRNA, shRNA and miRNA, gene replacement where the desired gene in the form of plasmids and viral vectors, are directly administered and finally gene editing based therapy where mutations are modified using specific nucleases such as zinc-finger nucleases (ZFNs), transcription activator-like effector nucleases (TALENs) and clustered regulatory interspaced short tandem repeats (CRISPR)/CRISPR-associated protein ...
Source: Cell Research - February 6, 2022 Category: Cytology Authors: Nilofer Sayed Prince Allawadhi Amit Khurana Vishakha Singh Umashanker Navik Sravan Kumar Pasumarthi Isha Khurana Anil Kumar Banothu Ralf Weiskirchen Kala Kumar Bharani 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

Versatile fully biodegradable dendritic nanotherapeutics
Biomaterials. 2022 Jan 3;281:121356. doi: 10.1016/j.biomaterials.2021.121356. Online ahead of print.ABSTRACTThe repeated administration of non-degradable dendrimers can lead to toxicity due to their bioaccumulation. Furthermore, in drug delivery applications, carrier stability can result in low biological performance due to insufficient intracellular cargo release. A novel family of versatile, biosafe, water-soluble, and fully biodegradable PEG-dendritic nanosystems is proposed, which overcomes the limitations of the most used dendrimers. Their novelty relies on the full and adjustable degradability thanks to the presence ...
Source: Biomaterials - January 15, 2022 Category: Materials Science Authors: Victoria Leiro Ana Patr ícia Spencer Nat ália Magalhães Ana Paula P êgo Source Type: research

Preparation and Preliminary Evaluation of Dual-functional Nanoparticles for MRI and siRNA Delivery
In conclusion, the visualized polycationic lipid siRNA delivery vehicle (LP-PEI1800-SPION) was successfully prepared in this experiment, which provides a research basis for further theranostics of liver cancer.
Source: Iranian Journal of Pharmaceutical Research - December 26, 2021 Category: Drugs & Pharmacology Source Type: research

A Nanoparticle-Conjugated Anti-TBK1 siRNA Induces Autophagy-Related Apoptosis and Enhances cGAS-STING Pathway in GBM Cells
CONCLUSION: The rGO-PEG could be an efficient system facilitating the delivery of specific siRNA. TBK1si/rGO-PEG could be a novel strategy for the treatment of GBM.PMID:34931127 | PMC:PMC8684524 | DOI:10.1155/2021/6521953
Source: Evidence-based Complementary and Alternative Medicine - December 21, 2021 Category: Complementary Medicine Authors: Shengchao Xu Xi Yan Lu Tang Gan Dai Chengke Luo Source Type: research

EZH2 as a new therapeutic target in brain tumors: Molecular landscape, therapeutic targeting and future prospects
Biomed Pharmacother. 2021 Dec 11;146:112532. doi: 10.1016/j.biopha.2021.112532. Online ahead of print.ABSTRACTBrain tumors are responsible for high mortality and morbidity worldwide. The brain tumor treatment depends on identification of molecular pathways involved in progression and malignancy. Enhancer of zeste homolog 2 (EZH2) has obtained much attention in recent years in field of cancer therapy due to its aberrant expression and capacity in modulating expression of genes by binding to their promoter and affecting methylation status. The present review focuses on EZH2 signaling in brain tumors including glioma, gliobla...
Source: Biomedicine and pharmacotherapy = Biomedecine and pharmacotherapie - December 15, 2021 Category: Drugs & Pharmacology Authors: Mahshid Deldar Abad Paskeh Atefeh Mehrabi Mohammad Hossein Gholami Amirhossein Zabolian Ehsan Ranjbar Hossein Saleki Adnan Ranjbar Mehrdad Hashemi Yavuz Nuri Ertas Kiavash Hushmandi Sepideh Mirzaei Milad Ashrafizadeh Ali Zarrabi Saeed Samarghandian Source Type: research

PEG-PEI/siROCK2 inhibits Aβ42-induced microglial inflammation via NLRP3/caspase 1 pathway
Conclusion PPSR inhibits Aβ42-induced microglial inflammation via NLRP3/caspase 1 pathway.
Source: NeuroReport - December 10, 2021 Category: Neurology Tags: Cellular, Molecular and Developmental Neuroscience Source Type: research