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Current Gene Therapy

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https://www.readbyqxmd.com/read/29189152/the-progress-of-gene-therapy-for-leber-s-optic-hereditary-neuropathy
#1
Yong Zhang, Zhen Tian, Jiajia Yuan, Chang Liu, Hong Li Liu, Si Qi Ma, Bin Li
Leber's Optic Hereditary Neuropathy (LHON) is a common cause of teenaged blindness in both eyes for which there is currently no effective treatment. In 1871, the German ophthalmologist Theodor Leber was the first to describe the clinical characteristics of his namesake disease, and through unremitting efforts over the past 100 years, researchers have continued to increase their understanding of LHON. In recent years, using gene therapy, several groups have obtained breakthroughs in the treatment of the disease...
November 29, 2017: Current Gene Therapy
https://www.readbyqxmd.com/read/29173172/therapeutic-applications-of-crispr-cas-for-duchenne-muscular-dystrophy
#2
Tatianna Wai Ying Wong, Ronald D Cohn
BACKGROUND: Duchenne muscular dystrophy (DMD) is an X-linked neuromuscular disease caused by the lack of dystrophin due to mutations in the DMD gene. Since dystrophin is essential in maintaining the integrity of the sarcolemmal membrane, the absence of the protein leads to muscle damage and DMD disease manifestation. Currently there is no cure with only symptomatic management available. OBJECTIVE: The most recent advancements in DMD therapies do not provide a permanent treatment for DMD...
November 21, 2017: Current Gene Therapy
https://www.readbyqxmd.com/read/29173171/new-developments-in-crispr-cas-based-functional-genomics-and-their-implications-for-research-using-zebrafish
#3
Sergey V Prykhozhij, Lucia Caceres, Jason N Berman
Genome editing using CRISPR/Cas9 has advanced very rapidly in its scope, versatility and ease of use. Zebrafish (Danio rerio) has been one of the vertebrate model species where CRISPR/Cas9 has been applied very extensively for many different purposes and with great success. In particular, disease modeling in zebrafish is useful for testing specific gene variants for pathogenicity in a preclinical setting. Here we describe multiple advances in diverse species and systems that can improve genome editing in zebrafish...
November 21, 2017: Current Gene Therapy
https://www.readbyqxmd.com/read/29173170/first-in-human-phase-1-crispr-gene-editing-cancer-trials-are-we-ready
#4
Francoise Baylis, Marcus McLeod
A prospective first-in-human Phase 1 CRISPR gene editing trial in the United States for patients with melanoma, synovial sarcoma, and multiple myeloma offers hope that gene editing tools may usefully treat human disease. An overarching ethical challenge with first-in-human Phase 1 clinical trials, however, is knowing when it is ethically acceptable to initiate such trials on the basis of safety and efficacy data obtained from pre-clinical studies. If the pre-clinical studies that inform trial design are themselves poorly designed - as a result of which the quality of pre-clinical evidence is deficient - then the ethical requirement of scientific validity for clinical research may not be satisfied...
November 21, 2017: Current Gene Therapy
https://www.readbyqxmd.com/read/29173169/crispr-cas9-gene-editing-from-basic-mechanisms-to-improved-strategies-for-enhanced-genome-engineering-in-vivo
#5
Jayme Salsman, Jean-Yves Masson, Alexandre Orthwein, Graham Dellaire
Targeted genome editing using the CRISPR/Cas9 technology is becoming a major area of research due to its high potential for the treatment of genetic diseases. Our understanding of this approach has expanded in recent years yet several new challenges have presented themselves as we explore the boundaries of this exciting new technology. Chief among these is improving the efficiency but also the preciseness of genome editing. The efficacy of CRISPR/Cas9 technology relies in part on the use of one of the major DNA repair pathways, Homologous recombination (HR), which is primarily active in S and G2 phases of the cell cycle...
November 21, 2017: Current Gene Therapy
https://www.readbyqxmd.com/read/29173168/crispr-cas9-editing-to-facilitate-and-expand-drug-discovery
#6
Francis Robert, Sidong Huang, Jerry Pelletier
INTRODUCTION: The ability of most laboratories to easily access CRISPR/Cas9 engineering tools has caused a revolution in biology. One of the areas that will continue to be impacted by genome editing is the drug discovery process. OBJECTIVE: CRISPR/Cas9 will not only serve to accelerate the drug discovery pipeline, but also streamline line it by identifying high-value targets, facilitating the validation of drug:target interactions and mechanisms of action, and stimulating the development of phenotype-based high throughput screens as alternatives to target-based assays...
November 21, 2017: Current Gene Therapy
https://www.readbyqxmd.com/read/29149824/the-role-of-gene-therapy-in-the-treatment-of-retinal-diseases-a-review
#7
Claudio Campa, Carla Enrica Gallenga, Elena Bolletta, Paolo Perri
Background: Gene therapy represents the therapeutic delivery of nucleic acid polymers into a patient's cells with the aim of treating an underlying disease. Over the past 2 decades this new therapy has made substantial progress owing to better understanding of the pathobiologic basis of various diseases coupled with growth of gene transfer biotechnologies. The eye, in particular, represents a suitable target for such therapy due to the immune privilege provided by the blood-ocular barrier, the ability to directly visualize, access and locally treat the cells and the minimal amount of vector needed given the size of this organ...
November 16, 2017: Current Gene Therapy
https://www.readbyqxmd.com/read/29141547/cardioprotective-effects-of-serca2a-overexpression-against-ischemia-reperfusion-induced-injuries-in-rats
#8
Yan Jian, Li-Li Tian, Lin-Hui Wang, Xiao-Dong Zhao, Jing-Wei Chen, Koji Murao, Wei Zhu, Liang Dong, Guo-Qing Wang, Wan-Ping Sun, Guo-Xing Zhang
AIMS: The aim of the present study is to assess how genetically increased Sarcoplasmic reticulum Ca2+-ATPase (Serca2a) expression affects cardiac injury after ischemia/reperfusion (I/R) exposure and the related mechanisms involved. METHODS AND RESULTS: Rats were subjected to left anterior descending coronary artery (LAD) occlusion for 30 min followed by a 24-hour reperfusion. Cardiac function analysis revealed that cardiac function dramatically improved in Serca2a transgenic rats (Serca2aTG) rats compared to wild type (WT) rats...
November 10, 2017: Current Gene Therapy
https://www.readbyqxmd.com/read/29034834/challenges-and-advances-in-gene-therapy-approaches-for-neurodegenerative-disorders
#9
Aneesh Donde, Philip C Wong, Liam L Chen
INTRODUCTION: The recent approval of Spinraza (nusinersen), an antisense oligonucleotide, by U.S. Food and Drug Administration to treat patients with spinal muscular atrophy, has reignited interests of researchers in designing and testing new gene therapy approaches to treat neurological disorders, in particular, to curb neurodegenerative diseases of the central nervous system which represent an everincreasing public health burden to today's society. CONCLUSION: This review highlights several key factors to be taken into consideration to design successful preclinical and clinical gene therapy experiments with respect to the vehicle of delivery and the route of administration to CNS-specific targets, with an additional focus on antisense oligonucleotide therapy and recent clinical trial developments...
October 13, 2017: Current Gene Therapy
https://www.readbyqxmd.com/read/28982328/effect-of-oral-nonionic-polymeric-micelles-delivered-parathyroid-hormone-cdna-on-bone-density-and-microarchitecture
#10
Sung-Hsiung Chen, Jih-Yang Ko, Fong-Fu Chou
INTRODUCTION: Parathyroid hormone (PTH) is an effective therapeutic agent for osteoporosis, but the treatment requires long-term daily injections. Oral gene delivery is a less invasive alternative to daily injections. The aim of this study is to investigate the effect of orally administered nonionic polymeric micelles of plasmid cDNA containing human cytomegalovirus promoter (PCMV)-PTH (1-34) plus EDTA on body mineral density and bone microstructure in ovariectomized rats. A total of 27 Spraque-Dawley female rats were subjected to a bilateral ovariectomy...
October 4, 2017: Current Gene Therapy
https://www.readbyqxmd.com/read/28982327/comparative-study-of-adeno-associated-virus-adenovirus-baculovirus-and-lentivirus-vectors-for-gene-therapy-of-the-eyes
#11
Giedrius Kalesnykas, Emmi Kokki, Laura Alasaarela, Hanna P Lesch, Timo Tuulos, Kati Kinnunen, Hannu Uusitalo, Kari Airenne, Seppo Ylä-Herttuala
Background The eye possesses unique anatomical features that make it a valuable target for gene therapy applications. Objective The aim of the current study was to compare transduction efficiency, safety and biodistribution of four viral vectors following intravitreal injection. Method Adenovirus (AdV), adeno-associated virus (AAV), baculovirus (BV) and lentivirus (LV) vectors encoding green fluorescent protein (GFP) were injected bilaterally intravitreally into adult C57BL/6OlaHsd mice. Control mice received saline...
October 3, 2017: Current Gene Therapy
https://www.readbyqxmd.com/read/28685672/micrornas-mediated-mmp-regulation-current-diagnostic-and-therapeutic-strategies-for-metabolic-syndrome
#12
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
#13
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
#14
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
#15
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
#16
EDITORIAL
Babak Mostaghaci
No abstract text is available yet for this article.
May 17, 2017: Current Gene Therapy
https://www.readbyqxmd.com/read/28494741/delivering-sirna-with-dendrimers-in-vivo-applications
#17
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
#18
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
#19
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
#20
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
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