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Current Gene Therapy

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https://www.readbyqxmd.com/read/29446741/whats%C3%A2-new-in-gene-therapy-of-hemophilia
#1
E Carlos Rodriguez-Merchan
BACKGROUND: Several methods have been investigated to effectively and safely transmit genes that stimulate cells to release therapeutic factor VIII (FVIII) and factor IX (FIX) into the circulation of people with hemophilia (PWH). OBJECTIVE: To review the role of gene therapy (GT) in PWH. METHODS: A Cochrane Library and PubMed (MEDLINE) search related to the role of GT in hemophilia was analyzed. RESULTS: The most promising vectors for hemophilia GT are adeno-associated virus (AAV) and lentivirus...
February 14, 2018: Current Gene Therapy
https://www.readbyqxmd.com/read/29446740/role-of-immune-cells-in-diabetic-kidney-disease
#2
Xiaoqian Yang, Shan Mou
Diabetic kidney disease (DKD) is one of the major complications of diabetes mellitus (DM) and is currently the most common cause of end-stage renal disease (ESRD) worldwide. Traditionally, DKD is considered a disease which has nothing to do with the immune system, and the pathogenesis is mainly characterized to be metabolic disturbance. Recent growing evidence indicates immunologic and inflammatory mechanisms in the development and progression of DKD. This overview of macrophages, dendritic cells, T lymphocytes, B lymphocytes, neutrophils and mast cells is closely involved in the pathologic process of DKD, with more emphasis on the leucocyte accumulation and related molecular mechanisms...
February 13, 2018: Current Gene Therapy
https://www.readbyqxmd.com/read/29446739/role-of-complement-properdin-in-renal-ischemia-reperfusion-injury
#3
Zinah Zwaini, Houyong Dai, Cordula Stover, Bin Yang
Renal ischemia-reperfusion injury (IRI) is one of the main causes of acute kidney injury (AKI), and may lead to chronic kidney disease. The high mortality rate of AKI has not changed in the last 5 decades due to non-recognition, nephrotoxin exposure, delayed diagnosis and lack of specific intervention. Complement activation plays important roles in IRI-induced AKI because of its association with immunity, inflammation, cell death and tissue repair. Nevertheless, the role of complement properdin, the sole positive regulator of the alternative pathway, in IRI-induced AKI has not been well defined...
February 13, 2018: Current Gene Therapy
https://www.readbyqxmd.com/read/29446738/how-do-dendritic-cells-play-the-role-in-ischemia-reperfusion-triggered-kidney-allograft-rejection
#4
Songjie Cai, Naotsugu Ichimaru, Shiro Takahara
In deceased donors, ischemia/reperfusion injury (IRI) is an important cause of allograft dysfunction. Prolonged cold and warm ischemia time leads to a high risk of early post-transplant complications, including acute and chronic rejection. Ischemia not only up-regulates inflammatory cytokines and chemokines, but also enhances the expression of MHC-class II and adhesion molecules on epithelial and dendritic cells. Moreover, the danger associated molecular patterns (DAMPs) released from stressed or dying cells, not only cause or amplify tissue inflammation and trigger tissue repair in response to IRI, but also act as adjuvants that enhance DC maturation and potentiate the adaptive immune response...
February 13, 2018: Current Gene Therapy
https://www.readbyqxmd.com/read/29446737/gene-modified-mesenchymal-stem-cell-based-therapy-in-renal-ischemia-reperfusion-injury
#5
Hongbo Xu, Cheng Chen, Linkun Hu, Jianquan Hou
Acute kidney injury (AKI) is a common syndrome in the clinic and has become a worldwide public health problem. Renal ischemia-reperfusion injury (IRI) is the most common cause of AKI. So far, effective treatment is still lacking for renal IRI, resulting in a high mortality rate of AKI. Mesenchymal stem cells (MSCs), considered as a promising candidate for tissue repair and regenerative medicine have aroused an increasing concern in recent years for the capacity of self-renewal and multi-lineage differentiation...
February 13, 2018: Current Gene Therapy
https://www.readbyqxmd.com/read/29446736/gene-therapy-in-kidney-transplantation-evidence-of-efficacy-and-future-directions
#6
Jiawei Li, Guisheng Qi, Guowei Tu, Cheng Yang, Ruiming Rong
Allograft loss remains a severe clinical problem after kidney transplantation. The molecular mechanism of graft loss is a complex process involving T and/or B cell activation, inflammation responses, autophagy and apoptosis. Since these pathways are involved in immune responses in kidney transplant rejection, application of genetic interference to inhibit specific pathways could present an effective targeted gene therapy method. Recent studies have successfully attempted to use gene therapy to target the key molecules involved in immune responses during transplantation...
February 13, 2018: Current Gene Therapy
https://www.readbyqxmd.com/read/29446735/epigenetic-regulation-of-regulatory-t-cells-in-kidney-disease-and-transplantation
#7
Yufeng Liu, Bo Peng, Shengdi Wu, Nuo Xu
Regulatory T (Treg) cells are a kind of immunosuppression cells, which have been used to treat autoimmune diseases and induce allograft tolerance in clinical trials. While Treg cells based therapy is a promising treatment for kidney diseases and an emerging concept for tolerance induction in renal transplantation, a better understanding of the functions and biology of Treg cells is needed to be able to optimally exploit them. Epigenetics regulation, which refers to potentially heritable alterations in gene expression without underlying changes of the nucleotide sequence, plays an important role in Treg cells induction and maintenance...
February 13, 2018: Current Gene Therapy
https://www.readbyqxmd.com/read/29446734/cx3cl1-cx3cr1-axis-as-the-therapeutic-potential-in-renal-diseases-friend-or-foe
#8
Quan Zhuang, Ke Cheng, Yingzi Ming
The fractalkine receptor chemokine (C-X3-C motif) receptor 1 (CX3CR1) and its highly selective ligand CX3CL1 mediate chemotaxis and adhesion of immune cells, which are involved in the pathogenesis and progression of numerous inflammatory disorders and malignancies. The CX3CL1/CX3CR1 axis has recently drawn attention as a potential therapeutic target because it is involved in the ontogeny, homeostatic migration, or colonization of renal phagocytes. We performed a Medline/PubMed search to detect recently published studies that explored the relationship between the CX3CL1/CX3CR1 axis and renal diseases and disorders, including diabetic nephropathy, renal allograft rejection, infectious renal diseases, IgA nephropathy, fibrotic kidney disease, lupus nephritis and glomerulonephritis, acute kidney injury and renal carcinoma...
February 13, 2018: Current Gene Therapy
https://www.readbyqxmd.com/read/29446733/current-understanding-of-inflammatory-responses-in-acute-kidney-injury
#9
Chao Hu, Yue Sheng, Zhijian Qian
Acute kidney injury has been a tough complex with increased mortality and morbidity. Inflammatory responses, including innate and adaptive immune responses, involve in the initiation and development of acute kidney injury, especially under the ischemic circumstances. Tubular cells and distinct immune cell subgroups play a critical role in the pathogenesis of inflammation. Current gene therapies show their benefits in renal repair. Here, we reviewed the renal inflammatory infiltration, inflammatory mediators, oxidative stress and potential signaling pathways, which give rise to the kidney diseases, in the mechanism of acute kidney injury...
February 13, 2018: Current Gene Therapy
https://www.readbyqxmd.com/read/29366415/dosage-and-passage-dependent-neuroprotective-effects-of-exosomes-derived-from-rat-bone-marrow-mesenchymal-stem-cells-an-in-vitro-analysis
#10
Chaitra Venugopal, Christopher Shamir, Sivapriya Senthilkumar, Janitri Venkatachala Babu, Peedikayil Kurien Sonu, Kusum Jain Nishtha, Kiranmai S Rai, K Shobha, Anandh Dhanushkodi
BACKGROUND: Neurodegenerative diseases comprise a group of disorders for which no treatment is available till date. Stem cell based therapy offers great hope and promise. However, stem cell transplantation is associated with certain disadvantages like poor targeted migration, engraftment and survival of the transplanted cells. MATERIAL & METHOD: Exosomes, a type of extracellular membrane vesicle released by all cell types including stem cells, offer an alternative to stem cell transplantation...
January 24, 2018: Current Gene Therapy
https://www.readbyqxmd.com/read/29357792/nonviral-delivery-systems-for-cancer-gene-therapy-strategies-and-challenges
#11
Gayong Shim, Dongyoon Kim, Quoc-Viet Le, Gyu Thae Park, Taekhyun Kwon, Yu-Kyoung Oh
Gene therapy has been receiving widespread attention due to its unique advantage in regulating the expression of specific target genes. In the field of cancer gene therapy, modulation of gene expression has been shown to decrease oncogenic factors in cancer cells or increase immune responses against cancer. Due to the macromolecular size and highly negative physicochemical features of plasmid DNA, efficient delivery systems are an essential ingredient for successful gene therapy. To date, a variety of nanostructures and materials have been studied as nonviral gene delivery systems...
January 19, 2018: Current Gene Therapy
https://www.readbyqxmd.com/read/29357791/neurodegenerative-disorders-treatment-the-microrna-role
#12
Barbara Ridolfi, Hanin Abdel-Haq
Neurodegenerative diseases such as Alzheimer's disease, Parkinson's disease, amyotrophic lateral sclerosis, Huntington's disease and prion disease are not timely and effectively treated using conventional therapies. This emphasizes the need for alternative therapeutic approaches. In this respect, gene-based therapies have been adopted as potentially feasible alternative therapies, where the microRNA (miRNA) approach has experienced a great explosion in recent years. Because miRNAs have been shown to be implicated in the pathogenesis of several diseases including neurodegenerative diseases, they are intensely studied as candidates for diagnostic and prognostic biomarkers, as predictors of drug response and as therapeutic agents...
January 19, 2018: Current Gene Therapy
https://www.readbyqxmd.com/read/29357790/a-new-era-for-hemoglobinopathies-more-than-one-curative-options
#13
Nikoletta Psatha, Penelope-Georgia Papayanni, Evangelia Yannaki
Hemoglobinopathies, including severe β-thalassemia and sickle cell disease, represent the most common monogenic disorders worldwide. Allogeneic hematopoietic stem cell transplantation (allo-HCT) is the only approved curative option for these syndromes, albeit limited to patients having a suitable donor. Gene therapy, by making use of the patient's own hematopoietic stem cells to introduce a normal copy of the β-globin gene by viral vectors, bridged the gap between the need for cure of patients with hemoglobinopathies and the lack of a donor, without incurring the immunological risks of allo-HSCT...
January 19, 2018: Current Gene Therapy
https://www.readbyqxmd.com/read/29303078/molecular-adjuvants-based-on-plasmids-encoding-protein-aggregation-domains-affect-bone-marrow-niche-homeostasis
#14
Maria Giovanna Sabbieti, Giovanna Lacava, Andrea Amaroli, Luigi Marchetti, Roberta Censi, Piera Di Martino, Dimitrios Agas
BACKGROUND: During last years, DNA vaccine immunogenicity has been optimized by the employment of co-stimulatory molecules and molecular adjuvants. It has been reported that plasmid (pATRex), encompassing the DNA sequence for the von Willebrand A (vWA/A) domain of the Anthrax Toxin Receptor-1 (ANTXR-1, alias TEM8, Tumor Endothelial Marker 8), acts as strong immune adjuvant by inducing formation of insoluble intracellular aggregates. Markedly, we faced with upsetting findings regarding the safety of pATRex as adjuvant since the aggregosome formation prompted to osteopenia in mice...
January 5, 2018: Current Gene Therapy
https://www.readbyqxmd.com/read/29189152/the-progress-of-gene-therapy-for-leber-s-optic-hereditary-neuropathy
#15
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
#16
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
#17
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
#18
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
#19
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
#20
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
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