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https://www.readbyqxmd.com/read/28743034/therapeutic-advances-in-musculoskeletal-aav-targeting-approaches
#1
REVIEW
Karen Bulaklak, Xiao Xiao
The use of recombinant adeno-associated viruses (rAAVs) is highly prevalent in musculoskeletal gene therapies due to their versatility, high transduction efficiency, natural tropism and vector genome persistence for years. As the largest organ in the body, treatment of skeletal muscle for widespread and sufficient therapeutic gene expression is highly challenging. In addition to disease-specific hurdles, vector genome loss, off-target gene transfer and immune responses to treatment can diminish the overall benefit of rAAV therapies...
July 22, 2017: Current Opinion in Pharmacology
https://www.readbyqxmd.com/read/28742067/long-term-microdystrophin-gene-therapy-is-effective-in-a-canine-model-of-duchenne-muscular-dystrophy
#2
Caroline Le Guiner, Laurent Servais, Marie Montus, Thibaut Larcher, Bodvaël Fraysse, Sophie Moullec, Marine Allais, Virginie François, Maeva Dutilleul, Alberto Malerba, Taeyoung Koo, Jean-Laurent Thibaut, Béatrice Matot, Marie Devaux, Johanne Le Duff, Jack-Yves Deschamps, Inès Barthelemy, Stéphane Blot, Isabelle Testault, Karim Wahbi, Stéphane Ederhy, Samia Martin, Philippe Veron, Christophe Georger, Takis Athanasopoulos, Carole Masurier, Federico Mingozzi, Pierre Carlier, Bernard Gjata, Jean-Yves Hogrel, Oumeya Adjali, Fulvio Mavilio, Thomas Voit, Philippe Moullier, George Dickson
Duchenne muscular dystrophy (DMD) is an incurable X-linked muscle-wasting disease caused by mutations in the dystrophin gene. Gene therapy using highly functional microdystrophin genes and recombinant adeno-associated virus (rAAV) vectors is an attractive strategy to treat DMD. Here we show that locoregional and systemic delivery of a rAAV2/8 vector expressing a canine microdystrophin (cMD1) is effective in restoring dystrophin expression and stabilizing clinical symptoms in studies performed on a total of 12 treated golden retriever muscular dystrophy (GRMD) dogs...
July 25, 2017: Nature Communications
https://www.readbyqxmd.com/read/28741493/derisking-drug-induced-carcinogenicity-for-novel-therapeutics
#3
REVIEW
Jonathan G Moggs, Timothy MacLachlan, Hans-Joerg Martus, Philip Bentley
Assessing the carcinogenic potential of innovative drugs spanning diverse therapeutic modalities and target biology represents a major challenge during drug development. Novel modalities, such as cell and gene therapies that involve intrinsic genetic modification of the host genome, require distinct approaches for identification of cancer hazard. We emphasize the need for customized weight-of-evidence cancer risk assessments based on mode of action that balance multiple options for preclinical identification of cancer hazard with appropriate labeling of clinical products and risk management plans...
August 2016: Trends in Cancer
https://www.readbyqxmd.com/read/28741454/nanovesicular-formulations-for-cancer-gene-therapy
#4
Lorena Tavano, Elisabetta Mazzotta, Rita Muzzalupo
In the last decades, gene therapy has become a novel therapeutic strategy for cancer treatment, including immunologic and molecular approaches. Among molecular avenue, the design of efficient and effective gene delivery systems, like cationic liposomes and niosomes, has been widely investigated and proposed as the most promising research area. The advantages of cationic vesicles rely on their natural ability to form complexes with anionic genetic molecules and deliver them into the cells via the endosomal pathway...
May 18, 2017: Current Pharmaceutical Design
https://www.readbyqxmd.com/read/28741230/new-therapeutic-strategies-for-lewy-body-dementias
#5
REVIEW
Latha Velayudhan, Dominic Ffytche, Clive Ballard, Dag Aarsland
This article reviews current treatment strategies and recent advances for the Lewy body dementias (LBDs). Current available symptom treatment strategies are based on monoaminergic, cholinergic and glutaminergic neurotransmitter systems. Relatively robust evidence exists for cholinesterase inhibitors for cognitive impairment in LBD and in Parkinson's disease for antidepressants, clozapine and recently pimavanserin for psychosis. interpidine (RVT 101) and nelotanserin are currently under investigation. Non-pharmacological interventions, such as cognitive stimulation, physical exercises and neuromodulation strategies, may be useful in Parkinson's disease but have not yet been tested in dementias...
September 2017: Current Neurology and Neuroscience Reports
https://www.readbyqxmd.com/read/28740654/a-novel-therapeutic-approach-for-esophageal-squamous-cell-carcinoma-suppressor-of-cytokine-signaling-1-gene-therapy
#6
EDITORIAL
Chang-Han Chen, Shau-Hsuan Li
No abstract text is available yet for this article.
June 2017: Journal of Thoracic Disease
https://www.readbyqxmd.com/read/28739283/the-pharmacology-of-gene-therapy
#7
EDITORIAL
Seppo Ylä-Herttuala
No abstract text is available yet for this article.
July 21, 2017: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/28737744/delivering-advanced-therapies-the-big-pharma-approach
#8
REVIEW
J Tarnowski, D Krishna, L Jespers, A Ketkar, R Haddock, J Imrie, S Kili
After two decades of focused development and some recent clinical successes, cell and gene therapy (CGT) is emerging as a promising approach to personalized medicines. Genetically engineered cells as a medical modality are poised to stand alongside or in combination with small molecule and biopharmaceutical approaches to bring new therapies to patients globally. Big pharma can play a vital role in industrializing CGT by focusing on diseases with high un-met medical need and compelling genetic evidence. Pharma should invest in manufacturing and supply chain solutions that deliver reproducible, high quality therapies at a commercially viable cost...
July 24, 2017: Gene Therapy
https://www.readbyqxmd.com/read/28737743/development-of-gene-therapies-lessons-from-nusinersen
#9
REVIEW
L Xu, I Irony, W W Bryan, B Dunn
The nusinersen development and approval process provide important lessons regarding the pathway to marketing approval for gene therapies. These lessons emphasize rigorous clinical trial design, flexibility in trial design and analysis, a collaborative effort with regular communications between the drug developer and the FDA, and use of FDA's expedited programs. These lessons are critical to the development of gene therapies for the treatment of serious or life-threatening rare diseases.Gene Therapy accepted article preview online, 24 July 2017...
July 24, 2017: Gene Therapy
https://www.readbyqxmd.com/read/28737023/monodispersed-bioactive-glass-nanoclusters-with-ultralarge-pores-and-intrinsic-exceptionally-high-mirna-loading-for-efficiently-enhancing-bone-regeneration
#10
Yumeng Xue, Yi Guo, Meng Yu, Min Wang, Peter X Ma, Bo Lei
Bioactive glass nanoparticles (BGNs) have attracted much attention in drug delivery and bone tissue regeneration, due to the advantages including biodegradation, high bone-bonding bioactivity, and facile large-scale fabrication. However, the wide biomedical applications of BGNs such as efficient gene delivery are limited due to their poor pore structure and easy aggregation. Herein, for the first time, this study reports novel monodispersed bioactive glass nanoclusters (BGNCs) with ultralarge mesopores (10-30 nm) and excellent miRNA delivery for accelerating critical-sized bone regeneration...
July 24, 2017: Advanced Healthcare Materials
https://www.readbyqxmd.com/read/28733131/preclinical-studies-for-a-phase-1-clinical-trial-of-autologous-hematopoietic-stem-cell-gene-therapy-for-sickle-cell-disease
#11
Fabrizia Urbinati, Jennifer Wherley, Sabine Geiger, Beatriz Campo Fernandez, Michael L Kaufman, Aaron Cooper, Zulema Romero, Filippo Marchioni, Lilith Reeves, Elizabeth Read, Barbara Nowicki, Elke Grassman, Shivkumar Viswanathan, Xiaoyan Wang, Roger P Hollis, Donald B Kohn
BACKGROUND AIMS: Gene therapy by autologous hematopoietic stem cell transplantation (HSCT) represents a new approach to treat sickle cell disease (SCD). Optimization of the manufacture, characterization and testing of the transduced hematopoietic stem cell final cell product (FCP), as well as an in depth in vivo toxicology study, are critical for advancing this approach to clinical trials. METHODS: Data are shown to evaluate and establish the feasibility of isolating, transducing with the Lenti/β(AS3)-FB vector and cryopreserving CD34(+) cells from human bone marrow (BM) at clinical scale...
July 18, 2017: Cytotherapy
https://www.readbyqxmd.com/read/28731526/drug-discovery-and-development-for-rare-genetic-disorders
#12
REVIEW
Wei Sun, Wei Zheng, Anton Simeonov
Approximately 7,000 rare diseases affect millions of individuals in the United States. Although rare diseases taken together have an enormous impact, there is a significant gap between basic research and clinical interventions. Opportunities now exist to accelerate drug development for the treatment of rare diseases. Disease foundations and research centers worldwide focus on better understanding rare disorders. Here, the state-of-the-art drug discovery strategies for small molecules and biological approaches for orphan diseases are reviewed...
July 21, 2017: American Journal of Medical Genetics. Part A
https://www.readbyqxmd.com/read/28731177/co%C3%A2-expression-of-axin-and-apc-gene-fragments-inhibits-colorectal-cancer-cell-growth-via-regulation-of-the-wnt-signaling-pathway
#13
Meili Xu, Xianling Liu, Yan Xu, Shicong Zhu, Yawen Gao
Adenomatous polyposis coli (APC) and Axin interactions serve an important role in colorectal cancer (CRC) pathogenesis. The aim of the present study was to assess the combined effects of Axin and APC co‑expression in CRC cells, and to determine the underlying mechanisms involved. SW480 cells were divided into the following groups: Untransfected (SW480 group), transfected with pEGFP‑N3plus pCS2‑MT (SW480/vector‑vector), transfected with pEGFP‑N3‑APC5 (SW480/APC5), and transfected with pEGFP‑N3‑APC5 pluspCS2‑MT‑Axin (SW480/APC5‑Axin)...
July 21, 2017: Molecular Medicine Reports
https://www.readbyqxmd.com/read/28731040/er-stress-and-the-unfolded-protein-response-in-neurodegeneration
#14
REVIEW
Claudio Hetz, Smita Saxena
The clinical manifestation of neurodegenerative diseases is initiated by the selective alteration in the functionality of distinct neuronal populations. The pathology of many neurodegenerative diseases includes accumulation of misfolded proteins in the brain. In physiological conditions, the proteostasis network maintains normal protein folding, trafficking and degradation; alterations in this network - particularly disturbances to the function of endoplasmic reticulum (ER) - are thought to contribute to abnormal protein aggregation...
July 21, 2017: Nature Reviews. Neurology
https://www.readbyqxmd.com/read/28731027/ptenp1-acts-as-a-cerna-to-regulate-pten-by-sponging-mir-19b-and-explores-the-biological-role-of-ptenp1-in-breast-cancer
#15
R-K Li, J- Gao, L-H Guo, G-Q Huang, W-H Luo
This study aimed to investigate role of long noncoding RNA PTENP1 regulating PTEN expression via miR-19b to affect breast cancer (BC) progression. We measured expressions of PTENP1, miR-19b and PTEN in 65 matched BC cancerous and noncancerous tissues by quantitative real-time fluorescence PCR (qRT-PCR) and investigated the biological effects of PTENP1 in BC MDA-MB-231 cells by several in vitro experiments including CCK8, wound healing, transwell and Annexin V-FITC/PI analysis. Besides, the competing endogenous RNA (ceRNA) activity of PTENP1 on miR-19b was detected by luciferase reporter assay, and the expressions of related genes and proteins were determined by western blot assay and qRT-PCR...
July 21, 2017: Cancer Gene Therapy
https://www.readbyqxmd.com/read/28731026/the-novel-gene-ha117-promotes-in-vitro-and-in-vivo-drug-resistance-in-mouse-colon-tumor-cells
#16
Y X Guo, Y H Xu, G H Zheng, X Q Jin
In this study, we characterized the role of HA117, a novel gene we previously identified, in drug resistance in vitro and in vivo. Briefly, CT26 cells expressing HA117 were obtained by infection with a recombinant adenovirus, and tested for drug sensitivity by methyl thiazolyl tetrazolium assay. In addition, the effect of HA117 on drug sensitivity was assessed in CT26 colon tumors transplanted into nude mice. In vitro, expression of HA117 increased the resistance of CT26 cells to 5-fluorouracil 2.75 times (P<0...
July 21, 2017: Cancer Gene Therapy
https://www.readbyqxmd.com/read/28729655/a-crispr-cas9-guidance-rna-screen-platform-for-hiv-provirus-disruption-and-hiv-aids-gene-therapy-in-astrocytes
#17
Zaohua Huang, Madahavan Nair
HIV/AIDS remains a major health threat despite significant advances in the prevention and treatment of HIV infection. The major reason is the inability of existing treatments to eradicate the multiple HIV reservoirs in the human body, including astrocytes in the human brain. CRISPR/Cas9 system is an emerging gene-editing technique with the potential to eliminate or disrupt HIV provirus in HIV reservoir cells, which may lead to a complete cure of HIV/AIDS. The key components of CRISPR/Cas9 are guide RNAs (gRNAs) which determine specific sequence targeting of DNAs...
July 20, 2017: Scientific Reports
https://www.readbyqxmd.com/read/28729230/x-linked-agammaglobulinaemia-outcomes-in-the-modern-era
#18
REVIEW
Ben Shillitoe, Andrew Gennery
Colonel Ogden Bruton reported X-Linked Agammaglobulinaemia in 1952 and treated the child with replacement immunoglobulin therapy. Over 60years later, the treatment for XLA has largely remained unchanged. Replacement immunoglobulin lacks the isotypes IgA and IgM, leading to concerns that patients continue to experience recurrent sinopulmonary tract infections and be at increased risk of bronchiectasis. There is potential hope of earlier diagnosis with newborn screening, and a potential cure for these patients, in the form of gene therapy...
July 17, 2017: Clinical Immunology: the Official Journal of the Clinical Immunology Society
https://www.readbyqxmd.com/read/28728523/structural-insights-into-the-binding-of-small-ligand-molecules-to-a-g-quadruplex-dna-located-in-the-hiv-1-promoter
#19
Petar M Mitrasinovic
Targeting guanine (G)-rich DNA sequences, folded into non-canonical G-quadruplex (G4) structures, by small ligand molecules is a promising strategy for gene therapy of various diseases. There is experimental proposal that, among eight studied ligands, nitidine chloride - NC and a benzo phenanthridine derivative - BPD have the highest binding affinities for such a sequence (5'-T(1)G(2)G(3)C(4)C(5)T(6)G(7)G(8)G(9)C(10)G(11)G(12)G(13)A(14)C(15)T(16)G(17)G(18)G(19)-3') in the HIV-1 promoter, indicating that an anti-HIV-1 prodrug may regulate the expression of the promoter...
July 20, 2017: Journal of Biomolecular Structure & Dynamics
https://www.readbyqxmd.com/read/28728449/efficiency-and-cytotoxicity-analysis-of-cationic-lipids-mediated-gene-transfection-into-ags-gastric-cancer-cells
#20
Nasrin Gharaati-Far, Mohammad Reza Tohidkia, Alireza Dehnad, Yadollah Omidi
In this effort, we provided comparative study on optimization of transfection conditions for AGS human gastric cancer cell line using two commercial non-liposomal cationic lipids. Using reporter vector pEGFP-N1, transfection efficiency of Attractene™ and X-tremeGENE HP™ transfection reagents in terms of cell densities and DNA/reagent ratios was determined in AGS cells by flow cytometry and fluorescence microscopy. In addition, influence of transfection reagents on direct cytotoxicity and cell viability was respectively, measured using lactate dehydrogenase (LDH) leakage and MTT assays...
July 20, 2017: Artificial Cells, Nanomedicine, and Biotechnology
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