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David M Ng, Amanda J Hooper, Matthew I Bellgard, John R Burnett
PURPOSE OF REVIEW: We review the role, utility and current status of patient registries for rare genetic lipid disorders. RECENT FINDINGS: The creation and maintenance of rare genetic lipid disorder patient registries is critical for disease monitoring, improving clinical best practice, facilitating research and enabling the development of novel therapeutics. An open-source disease registry platform, termed the Rare Disease Registry Framework, has been developed, optimized and deployed for homozygous familial hypercholesterolemia...
April 2018: Current Opinion in Lipidology
Feng Yin, Kuan Hu, Yangzi Chen, Mengying Yu, Dongyuan Wang, Qianqian Wang, Ken-Tye Yong, Fei Lu, Yongye Liang, Zigang Li
Since the successful exfoliation of graphene from graphite in 2004, graphene and graphene oxide (GO) have been considered the most promising two-dimensional (2D) nanomaterials with distinguished physical and chemical characteristics and have attracted great attention in many different fields. Graphene oxide is well-known for its distinct physiochemical properties and shows only minimal cytotoxicity compared to carbon nanotubes. Until now, only limited efforts have been invested in utilizing GO for gene therapy in pancreatic cancer treatments...
2017: Theranostics
Elisabeth Steinhagen-Thiessen, Erik Stroes, Handrean Soran, Colin Johnson, Philippe Moulin, Giorgio Iotti, Marco Zibellini, Bas Ossenkoppele, Michaela Dippel, Maurizio R Averna
A good understanding of the natural history of rare genetic lipid disorders is a pre-requisite for successful patient management. Disease registries have been helpful in this regard. Lipoprotein Lipase Deficiency (LPLD) is a rare, autosomal-recessive lipid disorder characterized by severe hypertriglyceridemia and a very high risk for recurrent acute pancreatitis, however, only limited data are available on its natural course. Alipogene tiparvovec (Glybera® ) is the first gene therapy to receive Marketing Authorization in the European Union; GENIALL (GENetherapy In the MAnagement of Lipoprotein Lipase Deficiency), a 15-year registry focusing on LPLD was launched in 2014 as part of its Risk Management Plan...
July 2017: Atherosclerosis
Rui Huang, Xinyu Yao, Yuan Chen, Xun Sun, Yunzhu Lin
Multidrug resistance (MDR) remains the major obstacle to the success of clinical cancer chemotherapy. P-glycoprotein (P-gp), encoded by the MDR1, is an important part with complex mechanisms associated with the MDR. In order to overcome the MDR of tumors, we in the present experimental design incorporated small interfering RNA (siRNA) targeting MDR1 gene and anticancer drug paclitaxel (PTX) into the solid lipid nanoparticles (SLNs) to achieve the combinational therapeutic effects of genetherapy and chemotherapy...
February 2016: Sheng Wu Yi Xue Gong Cheng Xue za Zhi, Journal of Biomedical Engineering, Shengwu Yixue Gongchengxue Zazhi
The treatment of congestive heart failure focusses on three steps: 1. Elimination of the precipitating cause or mechanism, and/or treatment of the underlying disease respectively. 2. Treatment of the failing heart syndrome itself. We shall concern ourselves with pharmacotherapy, omitting technical and surgical aspects. 3. Prophylactic treatment of complications, such as thromboembolism and arrhythmias. Drugs for the treatment of heart failure can be classified as follows: 1. Diuretics 2. Vasodilators 3. Neurohumoral Inhibitors 4...
May 1, 2000: Therapeutische Umschau. Revue Thérapeutique
Rosita Saraceno, Andrea Chiricozzi, Massimo Gabellini, Sergio Chimenti
BACKGROUND: Nanotechnology is a new branch of engineering consisting of the usage of nanoscale particles (100 nm and smaller). Nanomedicine is the application of nanoscale technologies for diagnostic and therapeutic purposes in medicine. Nanodermatology, nanotechnology applied to dermatology, represents one of the most advanced field for which an increasing interest, both economic and scientific, is rising. The skin is the first point of contact for a whole host of nanomaterials, ranging from topical preparations, articles of clothing and household products, to sporting goods and industrial manufactured goods...
February 2013: Skin Research and Technology
Su Q Li, He M Wang, Xiu F Cao
Esophageal carcinoma (EC) are characterized by dysregulation of microRNAs, which play an important roles as a posttranscriptional regulators in protein synthesis, and are involved in cellular processes, such as proliferation, apoptosis, and differentiation. Recently, altered miRNAs expression has been comprehensively studied in EC by high-throughput technology. Increased understanding of miRNAs target genes and their potential regulatory mechanisms have clarified the miRNAs activities and may provide exciting opportunities for cancer diagnosis and miRNA-based genetherapy...
December 2011: Biomarkers: Biochemical Indicators of Exposure, Response, and Susceptibility to Chemicals
Shao-yan Si, Shu-jun Song, Bing-xin Xu, Gang Zhao, Xiao-qing Tan, Jun-li Liu, Jian-zhong Zhang, Zhi-guo Liu
AIM: To construct recombinant co-expression adenovirus vector of SEA and CD80 genes regulated by mouse TERT(telomerase reverse transcriptase, TERT) promoter and to observe the expression of SEA and CD80 in the Hepa1-6 cells mediated by it. METHODS: Using AdEasy adenovirus system, the core promoter region of mTERT was subcloned to shuttle plasmid pShuttle2 and Myc-Max response element was inserted upstream of it to regulate the expression of SEA and CD80. The recombinant co-expression adenovirus vector of SEA and CD80 genes was constructed and named as Ad-MMRE-mTERT-BIS...
July 2011: Xi Bao Yu Fen Zi Mian Yi Xue za Zhi, Chinese Journal of Cellular and Molecular Immunology
R E Giunta, H-G Machens
Plastic surgery has passed through a very positive evolution in the last decades on the solid fundament of constantly developing academic plastic surgery. Aim of this paper is an objective evaluation of the current status of academic plastic surgery regarding research topics, currently available ressources and scientific outcome based on a questionnaire. The return rate of the questionnaire in academic departments was 92%. Main topics in research besides wound healing were topics from regenerative medicine such as tissue engineering, biomaterials, genetherapy and angiogenesis with the main focus on skin and fat tissues...
December 2009: Handchirurgie, Mikrochirurgie, Plastische Chirurgie
Kai Mei, Lian Wang, Ling Tian, Jingrui Yu, Zhixuan Zhang, Yuquan Wei
BACKGROUND: Interferon-gamma-inducible protein 10 (IP-10) is a potent inhibitor of tumor angiogenesis. It has been reported that the antiangiogenic therapy combined with chemotherapy has synergistic effects. METHODS: To elucidate the mechanisms of IP-10 gene combined with a chemotherapy agent, we intramuscularly injected pBLAST-IP-10 expression plasmid combined with gemcitabine into tumor-bearing mice. RESULTS: The proliferation of endothelial cells was effectively inhibited by IP-10 combined with gemcitabine in vitro...
2008: Journal of Experimental & Clinical Cancer Research: CR
Shao-yan Si, Pei-zhen Hu, Yang Huang, Xia Li, Wei Ge, Xiu-min Zhang, Yan-fang Sui, Jian-zhong Zhang, Ming Zhang
AIM: To observe the effects of hepatoma-targeting recombinant adenovirus vectors of staphylococcal enterotoxin A (SEA) and/or CD80 gene on hepatoma and to study its immunological mechanisms. METHODS: Using AdEasy adenovirus system, we constructed recombinant adenovirus vectors of SEA and/or CD80 gene driven by alpha-fetoprotein (AFP) enhancer I and promoter. After intratumoral therapy for the mice bearing subcutaneous xenograft hepatoma with the recombinant adenoviruses, SEA and/or CD80 mRNA and protein were detected by RT-PCR and Western blot...
March 2008: Xi Bao Yu Fen Zi Mian Yi Xue za Zhi, Chinese Journal of Cellular and Molecular Immunology
Noriaki Hosoya, Toshiyuki Miura, Ai Kawana-Tachikawa, Tomohiko Koibuchi, Tatsuo Shioda, Takashi Odawara, Tetsuya Nakamura, Yoshihiro Kitamura, Munehide Kano, Atsushi Kato, Mamoru Hasegawa, Yoshiyuki Nagai, Aikichi Iwamoto
Immuno-genetherapy using dendritic cells (DCs) can be applied to human immunodeficiency virus type 1 (HIV-1) infection. Sendai virus (SeV) has unique features such as cytoplasmic replication and high protein expression as a vector for genetic manipulation. In this study, we compared the efficiency of inducing green fluorescent protein (GFP) and HIV-1 gene expression in human monocyte-derived DCs between SeV and adenovirus (AdV). Human monocyte-derived DCs infected with SeV showed the maximum gene expression 24 hr after infection at a multiplicity of infection (MOI) of 2...
March 2008: Journal of Medical Virology
Xingyu Wang, Chunlan Wang, Yu Zhao
OBJECTIVE: To construct a replication-detective recombinant adinovirus including tne target gene human bone morphogenetic protein 4 (fragment hBMP-4). METHODS: The hBMP-4 gene fragment was cut down from pCS2(+)/hBMP-4, cloned into the eukaryotic expressive vector pcDNA 3.1(+), then subcloned into pShuttle-CMV and transformed into the competent E. coli BJ5183/p by electroporation. The resulting recombinant plasmid pAdE/ hBMP-4 was transformed into the packaging of the cell lines HEK293 to produce the replication-defective recombinant adenoviruses containing the hBMP-4 gene...
April 2007: Chinese Journal of Reparative and Reconstructive Surgery
J S Norris, A Bielawska, T Day, A El-Zawahri, S ElOjeimy, Y Hannun, D Holman, M Hyer, C Landon, S Lowe, J Y Dong, J McKillop, K Norris, L Obeid, S Rubinchik, M Tavassoli, S Tomlinson, C Voelkel-Johnson, X Liu
As of January 2005, there were 1020 gene therapy clinical trials ongoing worldwide with 675 or 66.2% devoted to cancer gene therapy. The majority are occurring in the US and Europe ( At the present time, to our knowledge there are no trials that employ gene delivery of Fas Ligand (FasL). As an important note, and in contrast to somatic cell therapy trials, there are no reported deaths due to therapeutic vector administration in any cancer gene therapy trial. That said, from our studies and from the published literature, the issue of gene delivery remains the major obstacle to successfully employing gene therapy for cancer treatment...
December 2006: Cancer Gene Therapy
Michael T Tetzlaff, Bin S Teh, Terry L Timme, Tetsuo Fujita, Takefumi Satoh, Ken-Ichi Tabata, Wei-Yuan Mai, Maria T Vlachaki, Robert J Amato, Dov Kadmon, Brian J Miles, Gustavo Ayala, Thomas M Wheeler, Estuardo Aguilar-Cordova, Timothy C Thompson, E Brian Butler
The advances in radiotherapy (3D-CRT, IMRT) have enabled high doses of radiation to be delivered with the least possible associated toxicity. However, the persistence of cancer (local recurrence after radiotherapy) despite these increased doses as well as distant failure suggesting the existence of micro-metastases, especially in the case of higher risk disease, have underscored the need for continued improvement in treatment strategies to manage local and micro-metastatic disease as definitively as possible...
February 2006: Technology in Cancer Research & Treatment
Na Ren, Feng Wang, Jun-qi Niu
OBJECTIVES: To explore, on the cell level, the possibility of using 10-23DNAzyme, as a new genetherapy in treating hepatitis B. METHODS: Phosthorothioate 10-23DRz (DRz-S) and 10-23DRz specific to HBV pre-C/C gene ORFA2031 were designed and synthesized, and the inhibition effects of 10-23DRz-S and 10-23DRz on the expression of HBV gene in HepG2 2.2.15 cells were observed. RESULTS: The expression of HBV gene was remarkably depressed after 2...
October 2005: Zhonghua Gan Zang Bing za Zhi, Zhonghua Ganzangbing Zazhi, Chinese Journal of Hepatology
G Wallin, G Lundell, J Tennvall
Anaplastic (giant cell) thyroid carcinoma (ATC), is one of the most aggressive malignancies in humans with a median survival time after diagnosis of 3-6 months. Death from ATC was earlier seen because of local growth and suffocation. ATC is uncommon, accounting for less than 5 % of all thyroid carcinomas. The diagnosis can be established by means of multiple fine needle aspiration biopsies, which are neither harmful nor troublesome for the patient. The cytological diagnosis of this high-grade malignant tumour is usually not difficult for a well trained cytologist...
2004: Scandinavian Journal of Surgery: SJS
Ning Li, Yu-Kang Yuan, Jun Wu
AIM: To construct adenovirus vector harboring CTLA4Ig-IRES 2-IkappaBalpha gene,and investigate its expression in ECV304 cells. METHODS: Recombinant adenovirus vector harboring CTLA4Ig-IRES 2-IkappaBalpha was constructed by homologous recombination in E.coli BJ5183. Then recombinant vector was packaged and propagated in 293 cells. ECV304 cells were infected with the recombinant adenovirus, and Western Blot was used to detect IkappaBalpha and CTLA4Ig protein expression in infected ECV304 cells...
January 2005: Xi Bao Yu Fen Zi Mian Yi Xue za Zhi, Chinese Journal of Cellular and Molecular Immunology
Cong-Mei Wu, Xiu-Yi Li, Tian-Hua Huang
AIM: To construct a pEgr-IFNgamma plasmid and to investigate its expression properties of interferon-gamma (INF-gamma) induced by irradiation and the effect of gene-radiotherapy on the growth of melanoma. METHODS: A recombined plasmid, pEgr-IFNgamma, was constructed and transfected into B16 cell line with lipofectamine. The expression properties of pEgr-IFNgamma were investigated by ELISA. Then, a B16 melanoma-bearing model was established in mice, and the plasmid was injected into the tumor tissue...
October 15, 2004: World Journal of Gastroenterology: WJG
Cong-Mei Wu, Xiu-Yi Li
OBJECTIVE: The pEgr-TNFalpha plasmid was constructed to investigate the effect of gene-radiotherapy on melanoma and host immune system. METHODS: pEgr-TNFalpha plasmids were constructed and injected into tumor tissue, 36 hours later, the tumors were given 20 Gy X-ray irradiation. Tumor growth at different timepoints was record and immunologic parameters were detected 15 days later. RESULTS: From 3 to 15 d after pEgr-TNFalpha gene-radiotherapy the tumor growth was significantly slower than irradiation or genetherapy alone...
March 2004: Zhonghua Zhong Liu za Zhi [Chinese Journal of Oncology]
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