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Therapy: Gene Therapy

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Total 490 results found since Jan 2013.

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

Lentivirus-mediated RNA interference targeting EBNA1 gene inhibits the growth of GT-38 cells in vitro and in vivo.
Authors: Wang J, Liang C, Meng F, Xu X, Wu Y, Lu L Abstract Epstein-Barr virus nuclear antigen 1 (EBNA1) is associated with the pathogenesis of Epstein-Barr virus-associated gastric carcinoma (EBVaGC). However, the function of EBNA1 in the growth of EBVaGC cells remains unclear. In the present study, the effects of silencing EBNA1, by RNA interference (RNAi), on the growth of EBVaGC cells were investigated in vitro and in vivo. A lentivirus-mediated RNAi targeting EBNA1 was transfected into the EBVaGC cell line GT-38. Reverse transcription-quantitative polymerase chain reaction (RT-qPCR), western blot analysis, MTT...
Source: Oncology Letters - August 14, 2019 Category: Cancer & Oncology Tags: Oncol Lett 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

Novel nanocarriers for silencing anti-phagocytosis CD47 marker in acute myeloid leukemia cells
Colloids Surf B Biointerfaces. 2022 Jun 1;217:112609. doi: 10.1016/j.colsurfb.2022.112609. Online ahead of print.ABSTRACTAcute myeloid leukemia (AML), a malignant disorder of Hematopoietic stem cells, can escape immunosurveillance by over expression of the cluster of differentiation 47 (CD47) marker, which functions as an inhibitory signal, suppressing phagocytosis by binding to signal regulatory protein α (SIRPα) on macrophages. AML is treated mainly by chemotherapy, which has drastic side effects and poor outcomes for the patients. Most AML patients develop drug resistance, so other methods to treat AML are highly requ...
Source: Colloids and Surfaces - June 6, 2022 Category: Biotechnology Authors: Eman M Hassan Shan Zou Source Type: research

Design and quality control of short interfering RNAs.
Authors: Abstract Review is devoted to analysis of design rules as well as quality controls in development of high effective and specific short interfering RNA (siRNA). Four crucial steps in development of such siRNAs are discussed: choice of target RNA, design of sense and antisense strands as well as assessment of activity and specificity of corresponding siRNAs. The special consideration is given to siRNA construction principles which are based on both structural and thermodynamical features and nucleotide composition of siRNAs as well as structural and thermodynamical properties of target RNA and features of ...
Source: Molekuliarnaia Biologiia - November 1, 2012 Category: Molecular Biology Tags: Mol Biol (Mosk) Source Type: research

Polymerase IV occupancy at RNA-directed DNA methylation sites requires SHH1
Nature advance online publication 01 May 2013. doi:10.1038/nature12178 Authors: Julie A. Law, Jiamu Du, Christopher J. Hale, Suhua Feng, Krzysztof Krajewski, Ana Marie S. Palanca, Brian D. Strahl, Dinshaw J. Patel & Steven E. Jacobsen DNA methylation is an epigenetic modification that has critical roles in gene silencing, development and genome integrity. In Arabidopsis, DNA methylation is established by DOMAINS REARRANGED METHYLTRANSFERASE 2 (DRM2) and targeted by 24-nucleotide small interfering RNAs (siRNAs) through a pathway termed RNA-directed DNA methylation (RdDM). This pathway requires two plant-specific RNA po...
Source: Nature AOP - May 1, 2013 Category: Research Authors: Julie A. LawJiamu DuChristopher J. HaleSuhua FengKrzysztof KrajewskiAna Marie S. PalancaBrian D. StrahlDinshaw J. PatelSteven E. Jacobsen Tags: Letter Source Type: research

Transforming growth factor-β and epithelial–mesenchymal transition are associated with pulmonary metastasis in adenoid cystic carcinoma
Conclusion: TGF-β1, phospho-Smad2 and β-catenin were significantly correlated with ACC metastasis. Blockade of TGF-β signaling by TβRIIDN or siRNA may offer potential gene therapies against pulmonary metastasis in patients with ACC.
Source: Oral Oncology - August 19, 2013 Category: Cancer & Oncology Authors: Ling Dong, Xi-Yuan Ge, Yi-Xiang Wang, Lin-Qian Yang, Sheng-Lin Li, Guang-Yan Yu, Yan Gao, Jia Fu Tags: Original articles Source Type: research

Structure and kinetics of lipid-nucleic acid complexes.
Abstract The structure and function of lipid-based complexes (lipoplexes) have been widely investigated as cellular delivery vehicles for nucleic acids-DNA and siRNA. Transfection efficiency in applications such as gene therapy and gene silencing has been clearly linked to the local, nano-scale organization of the nucleic acid in the vehicle, as well as to the global properties (e.g. size) of the carriers. This review focuses on both the structure of DNA and siRNA complexes with cationic lipids, and the kinetics of structure evolution during complex formation. The local organization of the lipoplexes is largely se...
Source: Advances in Colloid and Interface Science - January 28, 2014 Category: Chemistry Authors: Dan N, Danino D Tags: Adv Colloid Interface Sci Source Type: research

P5.07 * Targeting AKT2 signalling events: improving therapeutic outcomes through cancer stemness modulation
Conclusion: This study reveals the importance of AKT2 knockdown for E-cadherin restoration and metastatic potential decrease in mesenchymal BC cells. These results highlight AKT2 as a promising target for future gene therapy strategies concerning the prevention against CSC malignance and cancer recurrence.
Source: Annals of Oncology - March 20, 2015 Category: Cancer & Oncology Authors: Rafael, D., Gener, P., Pereira, L., Florindo, H., Schwartz, S., Videira, M. Tags: Poster session 5: New drugs/targets: signaling pathways Source Type: research

Chitosan as a non-viral co-transfection system in a cystic fibrosis cell line
This study shows proof-of-principle that co-transfection with chitosan might be an effective delivery system in a human CF cell line. It also offers a potential alternative to further develop therapeutic strategies for inherited disease treatments, such as CF. Graphical abstract
Source: International Journal of Pharmaceutics - February 22, 2016 Category: Drugs & Pharmacology Source Type: research

Efficient ferrocifen anticancer drug and Bcl-2 gene therapy using lipid nanocapsules on human melanoma xenograft in mouse
In conclusion, LNCs appeared to provide a promising tool for the co-encapsulation of a metal-based drug and siRNA. Graphical abstract
Source: Pharmacological Research - January 30, 2017 Category: Drugs & Pharmacology Source Type: research

Effect of inhibiting Beclin-1 expression on autophagy, proliferation and apoptosis in colorectal cancer.
In conclusion, Beclin-1 played an important role in regulating autophagy, proliferation and apoptosis in HCT116 and SW620 cells. The inhibition of Beclin-1 by RNAi suppressed the autophagic activity and proliferation, but promoted apoptosis in CRC cells. Beclin-1 was the new target of gene therapy for CRC. PMID: 28989537 [PubMed]
Source: Oncology Letters - October 12, 2017 Category: Cancer & Oncology Tags: Oncol Lett Source Type: research