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Current Gene Therapy

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https://www.readbyqxmd.com/read/28685672/micrornas-mediated-mmp-regulation-current-diagnostic-and-therapeutic-strategies-for-metabolic-syndrome
#1
Sharad Saxena, Aditi Jain, Vibha Rani
Metabolic syndrome (MS) is a global socioeconomic problem rapidly progressing in accordance with increasing body mass index (BMI) and age. It is a consortium of risk factors, such as dyslipidaemia, insulin resistance, leptin resistance, reduced adiponectin, glucose intolerance, hyperglycemia, and hypertension. Collectively, these factors accelerate the onset of type 2 diabetes mellitus, cardiovascular disease, stroke, and certain cancers such as breast, liver pancreatic, and colon cancer. Extracellular matrix (ECM) and basement membrane remodeling play a central role during pathogenesis of MS as they regulate diverse cell functions including proliferation, differentiation, and migration...
July 7, 2017: Current Gene Therapy
https://www.readbyqxmd.com/read/28595562/non-viral-delivery-systems-for-breast-cancer-gene-therapy
#2
Golnaz Vaseghi, Laleh Rafiee, Shaghayegh Haghjooy Javanmard
INTRODUCTION: The ever-evolving field of gene therapy promises several innovative treatments for cancer. Advances in genetic modification of tumor cells and micro environment have led to the development of more effective therapeutic strategies with fewer side effects. MATERIAL & METHODS: The development of effective delivery system challenges, remains. Non-viral vectors are interesting due to their bio-safety and their ability to transfer different types of nucleic acids...
June 6, 2017: Current Gene Therapy
https://www.readbyqxmd.com/read/28578643/glioblastoma-targeted-gene-therapy-based-on-pegfp-p53-loaded-superparamagnetic-iron-oxide-nanoparticles
#3
Touba Eslaminejad, Seyed Noureddin Nematollahi-Mahani, Mehdi Ansari
BACKGROUND: Bloodâ€"brain barrier (BBB) separates the neural tissue from circulating blood because of its high selectivity. This study focused on the in vitro application of magnetic nanoparticles to deliver Tp53 as a gene of interest to glioblastoma (U87) cells across a simulated BBB model that comprised KB cells. MATERIAL AND METHOD: After magnetic and non-magnetic nanoparticles were internalized by KB cells, their location in these cells was examined by transmission electron microscopy...
June 4, 2017: Current Gene Therapy
https://www.readbyqxmd.com/read/28552050/readthrough-of-scn5a-nonsense-mutations-r1623x-and-s1812x-question-gene-therapy-in-brugada-syndrome
#4
Siyong Teng, Jian Huang, Zhan Gao, Jie Hao, Yuejin Yang, Shu Zhang, Jielin Pu, Rutai Hui, Yongjian Wu, Zheng Fan
PURPOSE: Readthrough of nonsense mutation are used as gene-specific treatment in some genetic disease. Response to readthrough treatment is determined by readthrough efficiency of various nonsense mutations. In this manuscript, we aimed to explore the harmful effect of nonsense mutation suppressions. METHODS: HEK293 cells were transfected with two SCN5A (encode cardiac Na+ channel) nonsense mutations, p.R1623X and p.S1812X. We applied two readthrough-enhancing methods (either aminoglycosides or by a siRNA targeting eukaryotic release factor eRF3a (a GTPase that binds eRF1)) to suppress these SCN5A nonsense mutations...
May 28, 2017: Current Gene Therapy
https://www.readbyqxmd.com/read/28524008/editorial-smart-and-controllable-systems-for-gene-delivery
#5
EDITORIAL
Babak Mostaghaci
No abstract text is available yet for this article.
May 17, 2017: Current Gene Therapy
https://www.readbyqxmd.com/read/28494742/potential-gene-therapy-towards-treating-neurodegenerative-diseases-employing-polymeric-nanosystems
#6
Prachi Bangde, Sonal Atale, Anomitra Dey, Ashish Pandit, Prajakta Dandekar, Ratnesh Jain
Recent integrated approaches involving nanotechnology and gene therapy have accelerated development of efficient drug delivery to the central nervous system (CNS). Neurodegenerative disorders are closely associated with genetic inheritance and mutation. Nanotechnology has allowed effective engineering of various such polymeric structures. Moreover, availability of a wide array of polymeric materials has enabled fabrication of biocompatible and biodegradable delivery vehicles. Our review focuses on the ideal features and properties of polymeric nanoparticles that have enabled successful gene therapy for neurodegenerative disorders, as well as the challenges that are posing difficulties in their practical application...
May 10, 2017: Current Gene Therapy
https://www.readbyqxmd.com/read/28494741/delivering-sirna-with-dendrimers-in-vivo-applications
#7
Victoria Leiro, Sofia Duque Santos, Ana Paula Pêgo
Over the last decades, gene therapy has emerged as a pioneering therapeutic approach to treat or prevent several diseases. Among the explored strategies, the short-term silencing of protein coding genes mediated by siRNAs has a good therapeutic potential in a clinical setting. However, the widespread use of siRNA will require the development of clinically suitable, safe and effective vehicles with the ability to complex and deliver siRNA into target cells with minimal toxicity. Lately, dendrimers have gained considerable attention as non-viral vectors in nucleic acid delivery due to their unique structural characteristics (globular, well defined and highly branched structure, multivalency, low polydispersity and tunable nanosize), along with their relevant capacity to complex and protect nucleic acids in compact nanostructures, which can be functionalized with targeting moieties in order to get cell specificity...
May 10, 2017: Current Gene Therapy
https://www.readbyqxmd.com/read/28494740/nucleic-acids-based-nanotherapeutics-crossing-the-blood-brain-barrier
#8
Noha Nafee, Noha Gouda
The restless endeavors revealing the molecular pathways underlying many neurodegenerative diseases and brain tumors have paved the way for the introduction of the selective exogenous gene-based therapeutics. The implicated active biomolecules encompass mainly negatively-charged nucleic acids ranging from DNA, mRNA, non-coding RNAs (small-interfering RNA, siRNA, and microRNA, miRNA), to antisense oligonucleotides. They selectively interfere with the genes translational and/or transcriptional processes. Although many reviews previously addressed brain targeting, a thorough correlation between the molecular properties of these biomacromolecules, the nature of blood brain barrier (BBB) in the accompanying pathological condition, the intracellular targets, as well as the design of the delivery system which will transport the bioactive cargo to the target cells attempting efficient delivery to the active sites in the brain will be appraised...
May 10, 2017: Current Gene Therapy
https://www.readbyqxmd.com/read/28494739/gene-delivery-particle-engineering-strategies-for-shape-dependent-targeting-of-cells-and-tissues
#9
Kristen Lynn Kozielski, Metin Sitti
Successful gene delivery requires overcoming both systemic and intracellular obstacles before the nucleic acid cargo can successfully reach its tissue and subcellular target location. Non-viral mechanisms to enable targeting while avoiding off-target delivery have arisen via biological, chemical, and physical engineering strategies. Herein we will discuss the physical parameters in particle design that promote tissue- and cell-targeted delivery of genetic cargo. We will discuss systemic concerns, such as circulation, tissue localization, and clearance, as well as cell-scale obstacles, such as cellular uptake and nucleic acid packaging...
May 10, 2017: Current Gene Therapy
https://www.readbyqxmd.com/read/28494738/smart-and-controllable-raav-gene-delivery-carriers-in-progenitor-cells-for-human-musculoskeletal-regenerative-medicine-with-a-focus-on-the-articular-cartilage
#10
Ana Rey-Rico, Magali Cucchiarini
Cell therapy using mesenchymal stem cells (MSCs) is a powerful tool for the treatment of various diseases and injuries. Still, important limitations including the large amounts of cells required for application in vivo and the age-related decline in lifespan, proliferation, and potency may hinder the use of MSCs in patients. In this regard, gene therapy may offer strong approaches to optimize the use of MSCs for regenerative medicine. Diverse nonviral and viral gene vehicles have been manipulated to genetically modify MSCs, among which the highly effective and relatively safe recombinant adeno-associated viral (rAAV) vectors that emerged as the preferred gene delivery system to treat human disorders...
May 10, 2017: Current Gene Therapy
https://www.readbyqxmd.com/read/28494737/lipid-nanoparticles-as-potential-gene-therapeutic-delivery-systems-for-oral-administration
#11
Golnar Dorraj, Juan José Carreras, Hugo Núñez, Issam Abushammala, Ana Melero
Gene therapy has experimented an increasing attention in the last decades, due to its enormous potential applications in the medical field. It can be defined as the use of genes or genetic material (DNA, RNA, oligonucleotides) to treat or prevent a disease state, generally a genetic-based one. Other applications, like treating viral, bacterial or parasite infections or development of vaccines are gaining also interest. Efficient gene therapy is mainly dependent on the ability of the highly labile genetic material to reach the therapeutic target...
May 10, 2017: Current Gene Therapy
https://www.readbyqxmd.com/read/28494736/smart-micro-nano-robotic-systems-for-gene-delivery
#12
Alireza Pedram, Hossein Nejat Pishkenari
Small scale robotics have attracted growing attention for the prospect of targeting and accessing cell-sized sites, necessary for high precision biomedical applications and drug/gene delivery. The loss of controlled gene therapy, inducing systemic side effects and reduced therapeutic efficiency, can be settled utilizing these intelligent carriers. Newly proposed solutions for the main challenges of control, power supplying, gene release and final carrier extraction/degradation have shifted these smart miniature robots to the point of being employed for practical applications of transferring oligonucleotides (pDNA, siRNA, mRNA, etc...
May 10, 2017: Current Gene Therapy
https://www.readbyqxmd.com/read/28494735/perspectives-of-gene-therapies-in-autosomal-dominant-polycystic-kidney-disease
#13
Yuchen Xu, Ao Li, Guanqing Wu, Chaozhao Liang
Autosomal dominant polycystic kidney disease (ADPKD) is the most common inherited kidney disease in the clinic. The predominant clinical manifestation is bilateral and progressive cysts formation in the kidneys, impairs normal renal parenchyma, and ultimately leads to end-stage renal disease (ESRD). ADPKD is a heterogenic disease which is resulted from the mutations of PKD1 or PKD2 genes which encode polycystin-1 (PC1) and -2 (PC2), thereby multiple cell signaling pathways are involved. Although causative genes and aberrant signaling pathways have been investigated for decades, lack of effective and less side-effect treatment for the disease still perplex vast clinicians...
May 10, 2017: Current Gene Therapy
https://www.readbyqxmd.com/read/28494734/recent-advances-in-skin-penetration-enhancers-for-transdermal-gene-and-drug-delivery
#14
Morteza Amjadi, Babak Mostaghaci, Metin Sitti
There is a growing interest in transdermal delivery systems because of their noninvasive, targeted, and on-demand delivery of gene and drugs. However, efficient penetration of therapeutic compounds into the skin is still challenging largely due to the impermeability of the outermost layer of the skin, known as stratum corneum. Recently, there have been major research activities to enhance the skin penetration depth of pharmacological agents. This article reviews recent advances in the development of various strategies for skin penetration enhancement...
May 10, 2017: Current Gene Therapy
https://www.readbyqxmd.com/read/28494733/phage-mediated-gene-therapy
#15
Zeinab Hosseinidoust
Bacteriophages (bacterial viruses) have long been under investigation as vectors for gene therapy. Similar to other viral vectors, the phage coat proteins have evolved over millions of years to protect the viral genome from degradation post injection, offering protection for the valuable therapeutic sequence. However, what sets phage apart from other viral gene delivery vectors is their safety for human use and the relative ease by which foreign molecules can be expressed on the phage outer surface, enabling highly targeted gene delivery...
May 10, 2017: Current Gene Therapy
https://www.readbyqxmd.com/read/28403795/mesenchymal-stem-cell-derived-extracellular-vesicles-for-renal-repair
#16
Arash A Nargesi, Lilach O Lerman, Alfonso Eirin
Transplantation of autologous mesenchymal stem cells (MSCs) has been shown to attenuate renal injury and dysfunction in several animal models, and its efficacy is currently being tested in clinical trials for patients with renal disease. Accumulating evidence indicates that MSCs release extracellular vesicles (EVs) that deliver genes, microRNAs and proteins to recipient cells, acting as mediators of MSC paracrine actions. In this context, it is critical to characterize the MSC-derived EV cargo to elucidate their potential contribution to renal repair...
April 12, 2017: Current Gene Therapy
https://www.readbyqxmd.com/read/28382858/new-japanese-regulatory-frameworks-for-clinical-research-and-marketing-authorization-of-gene-therapy-and-cellular-therapy-products
#17
Sumimasa Nagai, Keiya Ozawa
In Japan, the Pharmaceuticals and Medical Devices Law was passed in 2014. In this new law, regenerative medical products were defined, and a conditional and term-limited approval system only for regenerative medical products was instituted. Gene therapy and adoptive cellular therapy are categorized as regenerative medical products. This law is intended for registration trials for marketing authorization. The Act on the Safety of Regenerative Medicine was also implemented in 2014. This act is intended for clinical research and medical practice involving processed cells other than registration trials...
April 6, 2017: Current Gene Therapy
https://www.readbyqxmd.com/read/28292253/the-adeno-associated-virus-a-safe-and-effective-vehicle-for-liver-specific-gene-therapy-of-inherited-and-non-inherited-diseases
#18
Kai Yan Mak, Indu G Rajapaksha, Peter W Angus, Chandana B Herath
The first human adeno-associated virus (AAV) was originally discovered in 1960s as a contaminant of adenovirus stocks preparation and thus it had not been of medical interest. Throughout last three decades AAV has gained popularity to be used in gene therapy, mainly due to its replicative defectiveness and lack of pathogenicity in human. In addition, the ability to mediate a stable and long-term expression in both non-dividing and dividing cells with specific tissue tropism makes AAV as one of the most promising candidates for therapeutic gene transfer to treat many genetic as well as non-genetic disorders...
March 14, 2017: Current Gene Therapy
https://www.readbyqxmd.com/read/28215147/approaches-to-optimize-gene-therapy-for-the-treatment-of-hematologic-malignancies-overcoming-the-obstacles
#19
Yingzhe Jiang, Bing Xia, Yizhuo Zhang, Wen Xu
Gene transfer and oncolytic viruses provide new therapeutic approaches for the treatment of hematologic malignancies. However, it is still too early to introduce gene delivery or oncolytic viruses into standard clinical protocol. It is very important to discuss the obstacles that gene transfer and oncolytic virotherapy face for the further clinical application for treatment of hematologic malignancies, and updating the advances made to overcome them. The major concerns in this review include the approaches of the development of immuno-stimulatory gene transfer mediated-vaccination for leukemia therapy, RNAi-based therapy for leukemia and enhancement of sensitivity of target malignant cells to virotherapy and alteration of host immune response to favor oncolytic viruses...
February 15, 2017: Current Gene Therapy
https://www.readbyqxmd.com/read/28155604/a-vector-based-on-the-chicken-hypersensitive-site-4-insulator-element-replicates-episomally-in-mammalian-cell
#20
Xi Zhang, Xiao-Yin Wang, Yan-Long Jia, Xiao Guo, Yan-Fang Wang, Tian-Yun Wang
Gene therapy in mammalian cells requires vectors exhibiting long-term stability and high expression. Episomal gene expression vectors offer a safe and attractive alternative to those that integrate into the host cell genome. In the present study, we developed a new episomal vector based on the insulator, chicken hypersensitive site 4 (cHS4). The cHS4 element was artificially synthesized, cloned into the pEGFP-C1 vector, and used to transfect Chinese hamster ovary (CHO) and human Chang liver cells. The stably transfected cell colonies were further cultured in either the presence or absence of G418 selection...
February 2, 2017: Current Gene Therapy
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