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Specialty: Molecular Biology
Therapy: Gene Therapy

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Total 32 results found since Jan 2013.

RNA interference (RNAi)-based therapeutics for treatment of rare neurologic diseases
Mol Aspects Med. 2022 Oct 15:101148. doi: 10.1016/j.mam.2022.101148. Online ahead of print.ABSTRACTAdvances in genome sequencing have greatly facilitated the identification of genomic variants underlying rare neurodevelopmental and neurodegenerative disorders. Understanding the fundamental causes of rare monogenic disorders has made gene therapy a possible treatment approach for these conditions. RNA interference (RNAi) technologies such as small interfering RNA (siRNA), microRNA (miRNA), and short hairpin RNA (shRNA), and other oligonucleotide-based modalities such as antisense oligonucleotides (ASOs) are being developed ...
Source: Molecular Medicine - October 18, 2022 Category: Molecular Biology Authors: Noelle D Germain Wendy K Chung Patrick D Sarmiere 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

MRI-Visible Nanovehicle for Efficient siRNA Delivery
Methods Mol Biol. 2021;2282:195-208. doi: 10.1007/978-1-0716-1298-9_13.ABSTRACTVisualizing siRNA delivery through medical imaging methods has drawn much attentions in recent gene therapy studies. Among them, iron oxide-based magnetic resonance imaging (MRI) is regarded as one of the most promising imaging modalities for its high spatial resolution as well as deep penetration and real-time properties. In this chapter, a detailed protocol of an amphiphilic superparamagnetic iron oxide (SPIO) nanovehicle-based siRNA delivery is described, mainly focusing on SPIO/siRNA complexes formation and characterization, in vitro and in ...
Source: Mol Biol Cell - April 30, 2021 Category: Molecular Biology Authors: Rongrong Jin Wencheng Zhu Gan Lin Gang Liu Hua Ai Source Type: research

Suppressing Sart1 to modulate macrophage polarization by siRNA-loaded liposomes: a promising therapeutic strategy for pulmonary fibrosis
Conclusion: Our study provides a valuable reference for modulating macrophage polarization and a promising strategy for the treatment of pulmonary fibrosis in clinical settings.
Source: Theranostics - January 15, 2021 Category: Molecular Biology Authors: Ting Pan, Qing Zhou, Kang Miao, Lei Zhang, Guorao Wu, Jun Yu, Yongjian Xu, Weining Xiong, Yong Li, Yi Wang Tags: Research Paper Source Type: research

pH-responsive DNA nanomicelles for chemo-gene synergetic therapy of anaplastic large cell lymphoma
Conclusion: DNMs co-loaded with Dox and ALK-specific siRNA exhibited significantly enhanced apoptosis of ALCL K299 cells in vitro and effectively inhibited tumor growth in vivo without obvious toxicity, providing a potential strategy in the development of nanomedicines for synergetic cancer therapy.
Source: Theranostics - October 3, 2020 Category: Molecular Biology Authors: Yuwei Li, Shuzhen Yue, Jingyu Cao, Chengzhan Zhu, Yixiu Wang, Xin Hai, Weiling Song, Sai Bi Tags: Research Paper Source Type: research

Mononuclear phagocyte system blockade improves therapeutic exosome delivery to the myocardium
Conclusions: Prior blocking of endocytosis of exosomes by macrophages with exosomeblocking successfully and efficiently improves the distribution of following exosometherapeutic in targeted organs, like the heart. The established two-step exosome delivery strategy (blocking the uptake of exosomes first followed by delivery of therapeutic exosomes) would be a promising method for gene therapy.
Source: Theranostics - July 3, 2020 Category: Molecular Biology Authors: Zhuo Wan, Lianbi Zhao, Fan Lu, Xiaotong Gao, Yan Dong, Yingxin Zhao, Mengying Wei, Guodong Yang, Changyang Xing, Li Liu Tags: Research Paper Source Type: research

RNA-based scaffolds for bone regeneration: application and mechanisms of mRNA, miRNA and siRNA
Globally, more than 1.5 million patients undergo bone graft surgeries annually, and the development of biomaterial scaffolds that mimic natural bone for bone grafting remains a tremendous challenge. In recent decades, due to the improved understanding of the mechanisms of bone remodeling and the rapid development of gene therapy, RNA (including messenger RNA (mRNA), microRNA (miRNA), and short interfering RNA (siRNA)) has attracted increased attention as a new tool for bone tissue engineering due to its unique nature and great potential to cure bone defects. Different types of RNA play roles via a variety of mechanisms in ...
Source: Theranostics - July 3, 2020 Category: Molecular Biology Authors: Qiuping Leng, Lini Chen, Yonggang Lv Tags: Review Source Type: research

Bioinspired tumor-homing nanoplatform for co-delivery of paclitaxel and siRNA-E7 to HPV-related cervical malignancies for synergistic therapy
Conclusion: Si/PNPs@HeLa, by integrating immune escape and tumor-homing ability, can serve as an efficient dual-drug delivery system to achieve precise treatment of cervical cancer through chemo-gene combined therapy.
Source: Theranostics - July 3, 2020 Category: Molecular Biology Authors: Cong Xu, Wan Liu, Yuan Hu, Weiping Li, Wen Di Tags: Research Paper Source Type: research

A method for gene knockdown in the retina using a lipid-based carrier.
Conclusions: This work supports the use of Invivofectamine 3.0 as a transfection agent for effective delivery of nucleic acids to the retina for gene function studies and as potential therapeutics. PMID: 32165826 [PubMed - in process]
Source: Molecular Vision - March 15, 2020 Category: Molecular Biology Tags: Mol Vis Source Type: research

A novel tumour suppressor lncRNA F630028O10Rik inhibits lung cancer angiogenesis by regulating miR-223-3p.
In this study, we carried out RNA sequencing (RNA-Seq) of tumour tissues isolated from LLC tumour-bearing mice treated with either Plasmodium yoelli (Py)-infected red blood cells or uninfected red blood cells. We found that F630028O10Rik (abbreviated as F63) is a novel lncRNA that was significantly up-regulated in tumours isolated from mice treated with Py-infected red blood cells compared to the control. By using gene silencing technique, F63 was found to inhibit both tumour Vascular Endothelial Growth Factor A (VEGFA) secretion and endothelial cells clone formation, migration, invasion and tube formation. Injection of ch...
Source: J Cell Mol Med - February 12, 2020 Category: Molecular Biology Authors: Qin L, Zhong M, Adah D, Qin L, Chen X, Ma C, Fu Q, Zhu X, Li Z, Wang N, Chen Y Tags: J Cell Mol Med Source Type: research

Supramolecular Assemblies of Peptides or Nucleopeptides for Gene Delivery
Using non-covalent interactions between nucleic acids (DNA, siRNA, miRNA, and mRNA) with peptides or nucleopeptides is a promising strategy to construct supramolecular assemblies for gene delivery and therapy. Comparing to conventional strategies for gene delivery, the assemblies of peptides or nucleopeptides provide several unique advantages: i) reversible interactions between the assemblies and the nucleic acids; ii) minimal immunogenicity; iii) biocompatibility. This field has advanced considerably in recent years so that it is worth summarizing the recent progresses and future challenges. In this review, we introduce t...
Source: Theranostics - June 13, 2019 Category: Molecular Biology Authors: Huaimin Wang, Zhaoqianqi Feng, Bing Xu Tags: Review Source Type: research

Otx2 enhances transdifferentiation of M üller cells‐derived retinal stem cells into photoreceptor‐like cells
In conclusion, Otx2 enhances transdifferentiation of MC‐RSCs into photoreceptor‐like cells and this is associated with the inhibition of Wnt signalling. Otx2 is a potential target f or gene therapy of retinal degenerative diseases.
Source: Journal of Cellular and Molecular Medicine - November 19, 2018 Category: Molecular Biology Authors: Yu Xiong, Hongpei Ji, Zhipeng You, Fei Yao, Rongrong Zhou, Weitao Song, Xiaobo Xia Tags: ORIGINAL ARTICLE Source Type: research

Otx2 enhances transdifferentiation of M üller cells-derived retinal stem cells into photoreceptor-like cells.
In conclusion, Otx2 enhances transdifferentiation of MC-RSCs into photoreceptor-like cells and this is associated with the inhibition of Wnt signalling. Otx2 is a potential target for gene therapy of retinal degenerative diseases. PMID: 30451368 [PubMed - as supplied by publisher]
Source: J Cell Mol Med - November 19, 2018 Category: Molecular Biology Authors: Xiong Y, Ji H, You Z, Yao F, Zhou R, Song W, Xia X Tags: J Cell Mol Med Source Type: research

Lentiviral ‑mediated inducible silencing of TLR4 attenuates neuropathic pain in a rat model of chronic constriction injury.
In conclusion, TLR4 may serve a significant role in neuropathy and the results of the present study provide an inducible lentivirus‑mediated siRNA against TLR4 that may serve as a potential novel strategy to be applied in gene therapy for NP in the future. PMID: 30365084 [PubMed - as supplied by publisher]
Source: Molecular Medicine Reports - October 27, 2018 Category: Molecular Biology Tags: Mol Med Rep Source Type: research