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https://www.readbyqxmd.com/read/29792508/effects-of-tgf-%C3%AE-overexpression-via-raav-gene-transfer-on-the-early-repair-processes-in-an-osteochondral-defect-model-in-minipigs
#1
Magali Cucchiarini, Ann-Kathrin Asen, Lars Goebel, Jagadeesh K Venkatesan, Gertrud Schmitt, David Zurakowski, Michael D Menger, Matthias W Laschke, Henning Madry
BACKGROUND: Application of the chondrogenic transforming growth factor beta (TGF-β) is an attractive approach to enhance the intrinsic biological activities in damaged articular cartilage, especially when using direct gene transfer strategies based on the clinically relevant recombinant adeno-associated viral (rAAV) vectors. PURPOSE: To evaluate the ability of an rAAV-TGF-β construct to modulate the early repair processes in sites of focal cartilage injury in minipigs in vivo relative to control (reporter lacZ gene) vector treatment...
May 1, 2018: American Journal of Sports Medicine
https://www.readbyqxmd.com/read/29790357/-control-editing-of-the-genome-within-reach-or-already-in-our-hands
#2
Radim Brdička, Radoslav Omelka
Although different genome editing tools have been around for decades, the recent emergence of cheap, quick, and accessible CRISPR/Cas9 technology has led to a revolution in this field. The technique has the potential to transform medicine from curative into preventive using a gene therapy. An application of genome editing has proven to be effective for both genetic and non-genetic (e.g. infectious) diseases. However, cancer and rare diseases treatment is at the forefront of interest. Concurrently, the legal and ethical frameworks should be discussed, especially as the technology moves towards a modification of the germ cells or embryos...
2018: Casopís Lékar̆ů C̆eských
https://www.readbyqxmd.com/read/29789638/human-carbonic-anhydrase-8-aav8-gene-therapy-inhibits-nerve-growth-factor-signaling-producing-prolonged-analgesia-and-anti-hyperalgesia-in-mice
#3
Gerald Z Zhuang, Udita Upadhyay, Xiaoying Tong, Yuan Kang, Diana M Erasso, Eugene S Fu, Konstantinos D Sarantopoulos, Eden R Martin, Tim Wiltshire, Luda Diatchenko, Shad B Smith, William Maixner, Roy C Levitt
Carbonic anhydrase-8 (Car8; murine gene symbol) is an allosteric inhibitor of inositol trisphosphate receptor-1 (ITPR1), which regulates neuronal intracellular calcium release. We previously reported that wild-type Car8 overexpression corrects the baseline allodynia and hyperalgesia associated with calcium dysregulation in the waddle (wdl) mouse due to a 19 bp deletion in exon 8 of the Car8 gene. In this report, we provide preliminary evidence that overexpression of the human wild-type ortholog of Car8 (CA8 WT ), but not the reported CA8 S100P loss-of-function mutation (CA8 MT ), inhibits nerve growth factor (NGF)-induced phosphorylation of ITPR1, TrkA (NGF high-affinity receptor), and ITPR1-mediated cytoplasmic free calcium release in vitro...
April 24, 2018: Gene Therapy
https://www.readbyqxmd.com/read/29789357/reactivation-of-%C3%AE-globin-in-adult-%C3%AE-yac-mice-after-ex-vivo-and-in-vivo-hematopoietic-stem-cell-genome-editing
#4
Chang Li, Nikoletta Psatha, Pavel Sova, Sucheol Gil, Hongjie Wang, Jiho Kim, Chandana Kulkarni, Cristina Valensisi, R David Hawkins, George Stamatoyannopoulos, André Lieber
Disorders involving β-globin gene mutations, mainly β-thalassemia and sickle cell disease, represent a major target for hematopoietic stem/progenitor cell (HSPC) gene therapy. This includes CRISPR/Cas9-mediated genome editing approaches in adult CD34+ cells aimed toward the re-activation of fetal γ-globin expression in red blood cells. Because models involving erythroid differentiation of CD34+ cells have limitations in assessing γ-globin re-activation, we focused on human β-globin locus transgenic (β-YAC) mice...
May 22, 2018: Blood
https://www.readbyqxmd.com/read/29788236/a-novel-adeno-associated-virus-capsid-with-enhanced-neurotropism-corrects-a-lysosomal-transmembrane-enzyme-deficiency
#5
Julie Tordo, Claire O'Leary, André S L M Antunes, Nuria Palomar, Patrick Aldrin-Kirk, Mark Basche, Antonette Bennett, Zelpha D'Souza, Hélène Gleitz, Annie Godwin, Rebecca J Holley, Helen Parker, Ai Yin Liao, Paul Rouse, Amir Saam Youshani, Larbi Dridi, Carla Martins, Thierry Levade, Kevin B Stacey, Daniel M Davis, Adam Dyer, Nathalie Clément, Tomas Björklund, Robin R Ali, Mavis Agbandje-McKenna, Ahad A Rahim, Alexey Pshezhetsky, Simon N Waddington, R Michael Linden, Brian W Bigger, Els Henckaerts
Recombinant adeno-associated viruses (AAVs) are popular in vivo gene transfer vehicles. However, vector doses needed to achieve therapeutic effect are high and some target tissues in the central nervous system remain difficult to transduce. Gene therapy trials using AAV for the treatment of neurological disorders have seldom led to demonstrated clinical efficacy. Important contributing factors are low transduction rates and inefficient distribution of the vector. To overcome these hurdles, a variety of capsid engineering methods have been utilized to generate capsids with improved transduction properties...
May 16, 2018: Brain: a Journal of Neurology
https://www.readbyqxmd.com/read/29787895/poly-amidoamine-pamam-dendrimer-mediated-delivery-of-drug-and-pdna-sirna-for-cancer-therapy
#6
REVIEW
Jun Li, Huamin Liang, Jing Liu, Ziyuan Wang
Poly (amidoamine) (PAMAM) dendrimers are well-defined, highly branched macromolecules with numerous active amine groups on the surface. Because of their unique properties, PAMAM dendrimers have steadily grown in popularity in drug delivery, gene therapy, medical imaging and diagnostic application. This review focuses on the recent developments on the application in PAMAM dendrimers as effective carriers for drug and gene (pDNA, siRNA) delivery in cancer therapy, including: a) PAMAM for anticancer drug delivery; b) PAMAM and gene therapy; c) PAMAM used in overcoming tumor multidrug resistance; d) PAMAM used for hybrid nanoparticles; and e) PAMAM linked or loaded in other nanoparticles...
May 19, 2018: International Journal of Pharmaceutics
https://www.readbyqxmd.com/read/29787711/tild-crispr-allows-for-efficient-and-precise-gene-knockin-in-mouse-and-human-cells
#7
Xuan Yao, Meiling Zhang, Xing Wang, Wenqin Ying, Xinde Hu, Pengfei Dai, Feilong Meng, Linyu Shi, Yun Sun, Ning Yao, Wanxia Zhong, Yun Li, Keliang Wu, Weiping Li, Zi-Jiang Chen, Hui Yang
The targeting efficiency of knockin sequences via homologous recombination (HR) is generally low. Here we describe a method we call Tild-CRISPR (targeted integration with linearized dsDNA-CRISPR), a targeting strategy in which a PCR-amplified or precisely enzyme-cut transgene donor with 800-bp homology arms is injected with Cas9 mRNA and single guide RNA into mouse zygotes. Compared with existing targeting strategies, this method achieved much higher knockin efficiency in mouse embryos, as well as brain tissue...
May 21, 2018: Developmental Cell
https://www.readbyqxmd.com/read/29787588/angiogenic-and-pleiotropic-effects-of-vegf165-and-hgf-combined-gene-therapy-in-a-rat-model-of-myocardial-infarction
#8
Pavel I Makarevich, Konstantin V Dergilev, Zoya I Tsokolaeva, Maria A Boldyreva, Evgeniy K Shevchenko, Evgeny V Gluhanyuk, Julia O Gallinger, Mikhail Yu Menshikov, Yelena V Parfyonova
Since development of plasmid gene therapy for therapeutic angiogenesis by J. Isner this approach was an attractive option for ischemic diseases affecting large cohorts of patients. However, first placebo-controlled clinical trials showed its limited efficacy questioning further advance to practice. Thus, combined methods using delivery of several angiogenic factors got into spotlight as a way to improve outcomes. This study provides experimental proof of concept for a combined approach using simultaneous delivery of VEGF165 and HGF genes to alleviate consequences of myocardial infarction (MI)...
2018: PloS One
https://www.readbyqxmd.com/read/29786563/cell-based-liver-therapies-past-present-and-future
#9
REVIEW
Valeria Iansante, Anil Chandrashekran, Anil Dhawan
Liver transplantation represents the standard treatment for people with an end-stage liver disease and some liver-based metabolic disorders; however, shortage of liver donor tissues limits its availability. Furthermore, whole liver replacement eliminates the possibility of using native liver as a possible target for future gene therapy in case of liver-based metabolic defects. Cell therapy has emerged as a potential alternative, as cells can provide the hepatic functions and engraft in the liver parenchyma...
July 5, 2018: Philosophical Transactions of the Royal Society of London. Series B, Biological Sciences
https://www.readbyqxmd.com/read/29785078/detection-of-hyper-conserved-regions-in-hepatitis-b-virus-x-gene-potentially-useful-for-gene-therapy
#10
Carolina González, David Tabernero, Maria Francesca Cortese, Josep Gregori, Rosario Casillas, Mar Riveiro-Barciela, Cristina Godoy, Sara Sopena, Ariadna Rando, Marçal Yll, Rosa Lopez-Martinez, Josep Quer, Rafael Esteban, Maria Buti, Francisco Rodríguez-Frías
AIM: To detect hyper-conserved regions in the hepatitis B virus (HBV) X gene ( HBX ) 5' region that could be candidates for gene therapy. METHODS: The study included 27 chronic hepatitis B treatment-naive patients in various clinical stages (from chronic infection to cirrhosis and hepatocellular carcinoma, both HBeAg-negative and HBeAg-positive), and infected with HBV genotypes A-F and H. In a serum sample from each patient with viremia > 3.5 log IU/mL, the HBX 5' end region [nucleotide (nt) 1255-1611] was PCR-amplified and submitted to next-generation sequencing (NGS)...
May 21, 2018: World Journal of Gastroenterology: WJG
https://www.readbyqxmd.com/read/29785047/rationally-designed-aav2-and-aavrh8r-capsids-provide-improved-transduction-in-the-retina-and-brain
#11
Jennifer A Sullivan, Lisa M Stanek, Michael J Lukason, Jie Bu, Shayla R Osmond, Elizabeth A Barry, Catherine R O'Riordan, Lamya S Shihabuddin, Seng H Cheng, Abraham Scaria
The successful application of adeno-associated virus (AAV) gene delivery vectors as a therapeutic paradigm will require efficient gene delivery to the appropriate cells in affected organs. In this study, we utilized a rational design approach to introduce modifications to the AAV2 and AAVrh8R capsids and the resulting variants were evaluated for transduction activity in the retina and brain. The modifications disrupted either capsid/receptor binding or altered capsid surface charge. Specifically, we mutated AAV2 amino acids R585A and R588A, which are required for binding to its receptor, heparan sulfate proteoglycans, to generate a variant referred to as AAV2-HBKO...
May 22, 2018: Gene Therapy
https://www.readbyqxmd.com/read/29784537/artificial-intelligence-a-joint-narrative-on-potential-use-in-pediatric-stem-and-immune-cell-therapies-and-regenerative-medicine
#12
REVIEW
Irena Sniecinski, Jerard Seghatchian
Artificial Intelligence (AI) reflects the intelligence exhibited by machines and software. It is a highly desirable academic field of many current fields of studies. Leading AI researchers describe the field as "the study and design of intelligent agents". McCarthy invented this term in 1955 and defined it as "the science and engineering of making intelligent machines". The central goals of AI research are reasoning, knowledge, planning, learning, natural language processing (communication), perception and the ability to move and manipulate objects...
May 9, 2018: Transfusion and Apheresis Science
https://www.readbyqxmd.com/read/29784433/fda-and-nist-collaboration-on-standards-development-activities-supporting-innovation-and-translation-of-regenerative-medicine-products
#13
Judith A Arcidiacono, Steven R Bauer, David S Kaplan, Clare M Allocca, Sumona Sarkar, Sheng Lin-Gibson
The development of standards for the field of regenerative medicine has been noted as a high priority by several road-mapping activities. Additionally, the U.S. Congress recognizes the importance of standards in the 21st Century Cure Act. Standards will help to accelerate and streamline cell and gene therapy product development, ensure the quality and consistency of processes and products, and facilitate their regulatory approval. Although there is general agreement for the need of additional standards for regenerative medicine products, a shared understanding of standards is required for real progress toward the development of standards to advance regenerative medicine...
May 18, 2018: Cytotherapy
https://www.readbyqxmd.com/read/29783206/-experimental-evaluation-of-the-therapeutic-potential-of-liposome-mediated-apoe3-gene-transfection-for-cerebral-atherosclerosis
#14
Vadym V Biloshytskyi, Alisa V Pachevska, Alina V Biloshytska, Valerii M Istoshyn, Maryna V Biloshytska, Olha B Shevnia
OBJECTIVE: Introduction: In recent years, a stroke has been the cause of high lethality, long-term and sustained disability, the problem of which is still far from being resolved. The root cause of stroke is atherosclerosis. The aim: Study of neurotrophic and neuroprotective functions of apoE in rat brain tissue in experimental atherosclerosis. PATIENTS AND METHODS: Materials and methods: The study included 60 sexually mature rats. The model of experimental atherosclerosis was created by Anichkov method...
2018: Wiadomości Lekarskie: Organ Polskiego Towarzystwa Lekarskiego
https://www.readbyqxmd.com/read/29783100/cd133-cells-derived-from-skeletal-muscles-of-duchenne-muscular-dystrophy-patients-have-a-compromised-myogenic-and-muscle-regenerative-capability
#15
Jinhong Meng, Francesco Muntoni, Jennifer Morgan
Cell-mediated gene therapy is a possible means to treat muscular dystrophies like Duchenne muscular dystrophy. Autologous patient stem cells can be genetically-corrected and transplanted back into the patient, without causing immunorejection problems. Regenerated muscle fibres derived from these cells will express the missing dystrophin protein, thus improving muscle function. CD133+ cells derived from normal human skeletal muscle contribute to regenerated muscle fibres and form muscle stem cells after their intra-muscular transplantation into an immunodeficient mouse model...
May 12, 2018: Stem Cell Research
https://www.readbyqxmd.com/read/29782834/high-throughput-dissection-of-aav-host-interactions-the-fast-and-the-curious
#16
REVIEW
Anne-Kathrin Herrmann, Dirk Grimm
Over fifty years after its initial description, Adeno-associated virus (AAV) remains a most exciting but also most elusive study object in basic or applied virology. On the one hand, its simple structure not only facilitates investigations into virus biology, but combined with the availability of numerous natural AAV variants with distinct infection efficiency and specificity also makes AAV a preferred substrate for engineering of gene delivery vectors. On the other hand, it is striking to witness a recent flurry of reports that highlight and partially close persistent gaps in our understanding of AAV virus and vector biology...
May 18, 2018: Journal of Molecular Biology
https://www.readbyqxmd.com/read/29782345/precision-surgery-for-obesity
#17
Zhiyong Dong, Sheikh Mohammed Shariful Islam, Enan Simms-Walker, Cunchuan Wang
Precision medicine is targeted towards improving the effectiveness of treatment, reducing the side effects of drugs and reducing medical costs. The application of precision surgery for obesity is a new concept that involves 2 stages: the first stage is to attain a precise obesity surgery, and the second stage is to achieve individualized obese gene therapy. In this article, we discuss the value of precision surgery for obesity, its stages and its future application to improve obesity surgery. Due to recent advancements in medical technologies, genetics, surgical and clinical research; precision surgery for obesity will lead the future of obesity surgery...
May 16, 2018: American Journal of Therapeutics
https://www.readbyqxmd.com/read/29781995/an-ecdysone-receptor-based-singular-gene-switch-for-deliberate-expression-of-transgene-with-robustness-reversibility-and-negligible-leakiness
#18
Seoghyun Lee, Minho Won, Ran Hee Hwang, Gang Min Hur, Hyunju Ro
Precise control of transgene expression is desirable in biological and clinical studies. However, because the binary feature of currently employed gene switches requires the transfer of two therapeutic expression units concurrently into a single cell, the practical application of the system for gene therapy is limited. To simplify the transgene expression system, we generated a gene switch designated as pEUI(+) encompassing a complete set of transgene expression modules in a single vector. Comprising of the GAL4 DNA-binding domain and modified EcR (GvEcR), a minimal VP16 activation domain fused with a GAL4 DNA-binding domain, as well as a modified Drosophila ecdysone receptor (EcR), the newly developed singular gene switch is highly responsive to the administration of a chemical inducer in a time- and dosage-dependent manner...
May 7, 2018: Journal of Visualized Experiments: JoVE
https://www.readbyqxmd.com/read/29781327/the-progress-of-aav-mediated-gene-therapy-in-neuromuscular-disorders
#19
Sara Aguti, Alberto Malerba, Haiyan Zhou
The well-defined genetic causes and monogenetic nature of many neuromuscular disorders, including Duchenne muscular dystrophy (DMD) and spinal muscular atrophy (SMA), present gene therapy as a prominent therapeutic approach. The novel variants of adeno-associated virus (AAV) can achieve satisfactory transduction efficiency of exogenous genes through the central nervous system and body-wide in skeletal muscle. Areas covered: In this review, we summarize the strategies of AAV gene therapy that are currently under preclinical and clinical evaluation for the treatment of degenerative neuromuscular disorders, with a focus on diseases such as DMD and SMA...
May 20, 2018: Expert Opinion on Biological Therapy
https://www.readbyqxmd.com/read/29781145/core-outcome-set-for-gene-therapy-in-haemophilia-results-of-the-corehem-multistakeholder-project
#20
A Iorio, M W Skinner, E Clearfield, D Messner, G F Pierce, M Witkop, S Tunis
BACKGROUND: Gene therapy trial results show potential to cure haemophilia A and haemophilia B. Securing broad access to a cure for a lifelong chronic disease is anticipated to face barriers at the individual and healthcare system levels, which can be partly mitigated by harmonized planning of clinical research studies. The aim of the coreHEM project was to determine the set of outcome measures required to evaluate efficacy, safety, comparative effectiveness and value of gene therapy for haemophilia...
May 20, 2018: Haemophilia: the Official Journal of the World Federation of Hemophilia
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