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Gene Therapy

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https://www.readbyqxmd.com/read/28440798/improved-gene-delivery-to-adult-mouse-spinal-cord-through-the-use-of-engineered-hybrid-adeno-associated-viral-serotypes
#1
J J Siu, N J Queen, W Huang, F Q Yin, X Liu, C Wang, D M McTigue, L Cao
Adeno-associated viral (AAV) vectors are often used in gene therapy for neurological disorders because of its safety profile and promising results in clinical trials. One challenge to AAV gene therapy is effective transduction of large numbers of the appropriate cell type, which can be overcome by modulating the viral capsid through DNA shuffling. Our previous study demonstrates that Rec2, among a family of novel engineered hybrid capsid serotypes (Rec1~4) transduces adipose tissue with far superior efficiency than naturally occurring AAV serotypes...
April 25, 2017: Gene Therapy
https://www.readbyqxmd.com/read/28440797/potential-mechanisms-of-microrna-141-3p-to-alleviate-chronic-inflammatory-pain-by-down-regulation-of-downstream-target-gene-hmgb1-in-vitro-and-in-vivo-studies
#2
W-S Shen, X-Q Xu, N-N Zhai, Z-S Zhou, J Shao, Y-H Yu
The present study aimed to investigate the potential role of microRNA-141-3p (miR-141-3p) in chronic inflammatory pain (CIP) by targeting HMGB1 gene. In the in vitro study, BV2 microglial cells were selected and assigned into blank, lipopolysaccharide (LPS), miR-141-3p mimics, mimics control, miR-141-3p inhibitor, inhibitor control, miR-141-3p mimics+LPS, mimics control+ LPS, miR-141-3p inhibitor+LPS, and inhibitor control+LPS groups. Ninety-six rats were randomly divided into 8 groups (12 rats in each group): blank control, model control, negative control (NC), miR-141-3p mimics+CFA, mimics control+CFA, HMGB1 shRNA+CFA, HMGB1 NC+CFA, and miR-141-3p mimics+HMGB1 shRNA+CFA groups...
April 25, 2017: Gene Therapy
https://www.readbyqxmd.com/read/28440796/the-importance-of-international-collaboration-for-rare-diseases-research-a-european-perspective
#3
REVIEW
D Julkowska, C P Austin, C M Cutillo, D Gancberg, C Hager, J Halftermeyer, A H Jonker, L P L Lau, I Norstedt, A Rath, R Schuster, E Simelyte, S van Weely
Over the last two decades, important contributions were made at national, European and international levels to foster collaboration into rare diseases research. The European Union (EU) has put much effort into funding rare diseases research, encouraging national funding organizations to collaborate together in the E-Rare programme, setting up European Reference Networks for rare diseases and complex conditions, and initiating the International Rare Diseases Research Consortium (IRDiRC) together with the National Institutes of Health in the USA...
April 25, 2017: Gene Therapy
https://www.readbyqxmd.com/read/28436941/insr-gene-polymorphisms-correlate-with-sensitivity-to-platinum-based-chemotherapy-and-prognosis-in-patients-with-epithelial-ovarian-cancer
#4
J-L Hu, X-L Hu, Q Han, A-Y Guo, C-J Wang, Y-Y Wen, S-D Cang
This study aimed to investigate the correlation between INSR gene polymorphisms on platinum-based chemotherapy sensitivity and prognosis in epithelial ovarian cancer (EOC). A total of 339 EOC patients receiving postoperative chemotherapy were recruited for the study. Tag single nucleotide polymorphism (tag SNP) of INSR gene was screened from HapMap combined with available literature. Frequency distribution of genotypes and alleles in INSR gene was sequenced by ABI3100-Avant. Compared with CC+GC genotype, INSR rs2252673 GG genotype and rs3745546 CC genotype showed less platinum-based chemotherapy sensitivity in EOC patients (OR=0...
April 24, 2017: Gene Therapy
https://www.readbyqxmd.com/read/28425480/efficient-cns-targeting-in-adult-mice-by-intrathecal-infusion-of-single-stranded-aav9-gfp-for-gene-therapy-of-neurological-disorders
#5
K Bey, C Ciron, L Dubreil, J Deniaud, M Ledevin, J Cristini, V Blouin, P Aubourg, M-A Colle
Adeno-associated virus (AAV) gene therapy constitutes a powerful tool for the treatment of neurodegenerative diseases. While AAVs are generally administered systemically to newborns in preclinical studies of neurological disorders, in adults the maturity of the blood-brain barrier (BBB) must be considered when selecting the route of administration. Delivery of AAVs into the cerebrospinal fluid (CSF) represents an attractive approach to target the central nervous system (CNS) and bypass the BBB. In this study, we investigated the efficacy of intra-CSF delivery of a single-stranded (ss) AAV9-CAG-GFP vector in adult mice via intracisternal (iCist) or intralumbar (it-Lumb) administration...
April 20, 2017: Gene Therapy
https://www.readbyqxmd.com/read/28346436/detailed-comparison-of-retroviral-vectors-and-promoter-configurations-for-stable-and-high-transgene-expression-in-human-induced-pluripotent-stem-cells
#6
D Hoffmann, J W Schott, F K Geis, L Lange, F-J Müller, D Lenz, D Zychlinski, D Steinemann, M Morgan, T Moritz, A Schambach
Correction of patient-specific induced pluripotent stem cells (iPSC) upon gene delivery through retroviral vectors offers new treatment perspectives for monogenetic diseases. Gene-modified iPSC clones can be screened for safe integration sites and differentiated into transplantable cells of interest. However, the current bottleneck is epigenetic vector silencing. In order to identify the most suitable retroviral expression system in iPSC, we systematically compared vectors from different retroviral genera, different promoters and their combination with ubiquitous chromatin opening elements (UCOE), and several envelope pseudotypes...
April 20, 2017: Gene Therapy
https://www.readbyqxmd.com/read/28414303/development-of-potent-class-ii-transactivator-gene-delivery-systems-capable-of-inducing-de-novo-mhc-ii-expression-in-human-cells-in-vitro-and-ex-vivo
#7
M de Lucena Palma, P Duangkhae, B Douradinha, I F T Viana, P O Rigato, R Dhalia, R B Mailliard, S M Barratt-Boyes, E J M do Nascimento, T M Oshiro, A J da Silva Duarte, E T de A Marques
Class II Transactivator (CIITA) induces transcription of MHC II genes and can potentially be used to improve genetic immunotherapies by converting non-immune cells into cells capable of presenting antigens to CD4(+) T cells. However, CIITA expression is tightly controlled and it remains unclear whether distinct non-immune cells differ in this transactivator regulation. Here we describe the development of gene delivery systems capable of promoting the efficient CIITA expression in non-immune cell lines and in primary human cells of an ex vivo skin explant model...
April 17, 2017: Gene Therapy
https://www.readbyqxmd.com/read/28387759/characterization-of-retroviral-infectivity-and-superinfection-resistance-during-retrovirus-mediated-transduction-of-mammalian-cells
#8
J Liao, Q Wei, J Fan, Y Zou, D Song, J Liu, F Liu, C Ma, X Hu, L Li, Y Yu, X Qu, L Chen, X Yu, Z Zhang, C Zhao, Z Zeng, R Zhang, S Yan, T Wu, X Wu, Y Shu, J Lei, Y Li, W Zhang, J Wang, R R Reid, M J Lee, W Huang, J M Wolf, T-C He, J Wang
Retroviral vectors including lentiviral vectors are commonly-used tools to stably express transgenes or RNA molecules in mammalian cells. Their utilities are roughly divided into two categories, stable overexpression of transgenes and RNA molecules, which requires maximal transduction efficiency, or functional selection with retrovirus-based libraries, which takes advantage of retroviral superinfection resistance. However, the dynamic features of retrovirus-mediated transduction are not well-characterized. Here, we engineered two MSCV-based retroviral vectors expressing dual fluorescence proteins and antibiotic markers and analyzed virion production efficiency and virion stability, dynamic infectivity and superinfection resistance in different cell types, and strategies to improve transduction efficiency...
April 7, 2017: Gene Therapy
https://www.readbyqxmd.com/read/28368370/mnsod-mediated-by-hsv-vectors-in-the-periaqueductal-gray-suppresses-morphine-withdrawal-in-rats
#9
T Iida, H Yi, S Liu, D Ikegami, W Zheng, Q Liu, K Takahashi, Y Kashiwagi, W F Goins, J C Glorioso, S Hao
Morphine appears to be the most active metabolite of heroin, therefore, the effects of morphine are important in understanding the ramifications of heroin abuse. Opioid physical dependence (withdrawal response) may have very long-lasting effects on the motivation for reward, including the incubation of cue-induced drug-seeking behavior. However, the exact mechanisms of morphine withdrawal (MW) are not clear yet, and its treatment remains elusive. Periaqueductal gray (PAG) is one of the important sites in the pathogenesis of MW...
April 3, 2017: Gene Therapy
https://www.readbyqxmd.com/read/28346435/aav9-mediated-engineering-of-autotransplanted-kidney-of-non-human-primates
#10
S Tomasoni, P Trionfini, N Azzollini, L Zentilin, M Giacca, S Aiello, L Longaretti, E Cozzi, N Baldan, G Remuzzi, A Benigni
Ex vivo gene transfer to the graft before transplantation is an attractive option for circumventing systemic side effects of chronic anti-rejection therapy. Gene delivery of the immunomodulatory protein CTLA4-Ig prevented chronic kidney rejection in a rat model of allotransplantation without the need for systemic immunosuppression. Here, we generated AAV2 and AAV9 vectors encoding for LEA29Y, an optimized version of CTLA4-Ig. Both LEA29Y vectors were equally efficient for reducing T-cell proliferation in vitro...
March 27, 2017: Gene Therapy
https://www.readbyqxmd.com/read/28346434/optimization-of-adeno-associated-virus-vector-mediated-gene-transfer-to-the-respiratory-tract
#11
F Kurosaki, R Uchibori, N Mato, Y Sehara, Y Saga, M Urabe, H Mizukami, Y Sugiyama, A Kume
An efficient adeno-associated virus (AAV) vector was constructed for the treatment of respiratory diseases. AAV serotypes, promoters, and routes of administration potentially influencing the efficiency of gene transfer to airway cells were examined in the present study. Among the nine AAV serotypes (AAV1-9) screened in vitro and four serotypes (AAV1, 2, 6, 9) evaluated in vivo, AAV6 showed the strongest transgene expression. As for promoters, the cytomegalovirus (CMV) early enhancer/chicken β-actin (CAG) promoter resulted in more robust transduction than the CMV promoter...
March 27, 2017: Gene Therapy
https://www.readbyqxmd.com/read/28218744/live-cell-imaging-to-compare-the-transfection-and-gene-silencing-efficiency-of-calcium-phosphate-nanoparticles-and-a-liposomal-transfection-agent
#12
S Chernousova, M Epple
The processing of DNA (for transfection) and siRNA (for gene silencing), introduced into HeLa cells by triple-shell calcium phosphate nanoparticles, was followed by live-cell imaging. For comparison, the commercial liposomal transfection agent Lipofectamine(TM) was used. The cells were incubated with these delivery systems, carrying either eGFP-encoding DNA or siRNA against eGFP. In the latter case, HeLa cells which stably expressed eGFP were used. The expression of eGFP started after 5 h in the case of nanoparticles and after 4 h in the case of Lipofectamine...
February 20, 2017: Gene Therapy
https://www.readbyqxmd.com/read/28165447/an-aavrh10-cag-cyp21-ha-vector-allows-persistent-correction-of-21-hydroxylase-deficiency-in-a-cyp21-mouse-model
#13
M Perdomini, C Dos Santos, C Goumeaux, V Blouin, P Bougnères
The treatment of severe forms of 21-hydroxylase deficiency (21OHD) remains unsatisfactory in many respects. As a monogenic disease caused by loss-of-function mutations, 21OHD is a potential candidate for a gene therapy (GT) approach. The first step of GT is to demonstrate positive effects of the therapeutic vector in the Cyp21(-/--) mouse model. Thus we tested the adrenal tropism of an AAVrh10-CAG-GFP vector ('GFP vector') then attempted to correct the phenotypic and biochemical alterations in Cyp21(-/-) mice using an AAVrh10-CAG-humanCYP21A2-HA vector ('CYP21 vector')...
February 6, 2017: Gene Therapy
https://www.readbyqxmd.com/read/28300083/mr-guided-parenchymal-delivery-of-adeno-associated-viral-vector-serotype-5-in-non-human-primate-brain
#14
L Samaranch, B Blits, W San Sebastian, P Hadaczek, J Bringas, V Sudhakar, M Macayan, P J Pivirotto, H Petry, K S Bankiewicz
The present study was designed to characterize transduction of non-human primate brain and spinal cord with AAV5 viral vector after parenchymal delivery. AAV5-CAG-GFP (1 × 10(13) vector genomes per milliliter (vg ml(-1))) was bilaterally infused either into putamen, thalamus or with the combination left putamen and right thalamus. Robust expression of GFP was seen throughout infusion sites and also in other distal nuclei. Interestingly, thalamic infusion of AAV5 resulted in the transduction of the entire corticospinal axis, indicating transport of AAV5 over long distances...
April 2017: Gene Therapy
https://www.readbyqxmd.com/read/28276446/systemic-injection-of-aav9-gdnf-provides-modest-functional-improvements-in-the-sod1-g93a-als-rat-but-has-adverse-side-effects
#15
G M Thomsen, M Alkaslasi, J-P Vit, G Lawless, M Godoy, G Gowing, O Shelest, C N Svendsen
Injecting proteins into the central nervous system that stimulate neuronal growth can lead to beneficial effects in animal models of disease. In particular, glial cell line-derived neurotrophic factor (GDNF) has shown promise in animal and cell models of Parkinson's disease, Huntington's disease and amyotrophic lateral sclerosis (ALS). Here, systemic AAV9-GDNF was delivered via tail vein injections to young rats to determine whether this could be a safe and functional strategy to treat the SOD1(G93A) rat model of ALS and, therefore, translated to a therapy for ALS patients...
April 2017: Gene Therapy
https://www.readbyqxmd.com/read/28221337/modulation-of-digital-flexor-tendon-healing-by-vascular-endothelial-growth-factor-gene-transfection-in-a-chicken-model
#16
W F Mao, Y F Wu, Q Q Yang, Y L Zhou, X T Wang, P Y Liu, J B Tang
A major challenge in tendon injury is the weak intrinsic healing capacity of tendon that may cause rupture of the repair after surgery. Growth factors are believed to be critical during tendon healing. This study aimed to investigate the effects of vascular endothelial growth factor (VEGF) genes delivered by adeno-associated virus (AAV) vectors on tendon healing and molecular events involved in a chicken model. A total of 128 deep flexor tendons in the long toes of chickens were completely transected and injected with 2 × 10(9) particles of AAV2-VEGF or saline before surgically repaired...
April 2017: Gene Therapy
https://www.readbyqxmd.com/read/28206989/il-4-mediated-by-hsv-vector-suppresses-morphine-withdrawal-response-and-decreases-tnf%C3%AE-nr2b-and-pc-ebp%C3%AE-in-the-periaqueductal-gray-in-rats
#17
H Yi, T Iida, S Liu, D Ikegami, Q Liu, A Iida, D A Lubarsky, S Hao
Chronic opiates induce the development of physical dependence. Opioid physical dependence characterized by withdrawal symptoms, may have very long-lasting effects on the motivation for reward, including the incubation of cue-induced drug-seeking behavior. Elucidation of the mechanisms involved in physical dependence is crucial to developing more effective treatment strategies for opioid dependence. Chronic morphine induces production of proinflammatory cytokines in regional-specific sites of the brain. Interleukin-4 (IL-4) is a prototypical anti-inflammatory cytokine that globally suppresses proinflammatory cytokines...
April 2017: Gene Therapy
https://www.readbyqxmd.com/read/28150697/neutralizing-antibodies-against-adeno-associated-viruses-in-sj%C3%A3-gren-s-patients-implications-for-gene-therapy
#18
A Corden, B Handelman, H Yin, A Cotrim, I Alevizos, J A Chiorini
One potential setback to the use of gene therapy for the treatment of Sjögren's syndrome is the presence of neutralizing antibodies (nAb) against adeno-associated virus (AAV) serotypes. In order to evaluate the efficacy of this treatment option, nAb titers were measured in both healthy individuals and Sjögren's patients. Several serotypes with known transduction activity in mouse salivary glands were tested and only AAV5 showed a statistically significant change in the prevalence of nAbs between Sjögren's and healthy participants...
April 2017: Gene Therapy
https://www.readbyqxmd.com/read/28079862/extraneuronal-pathology-in-a-canine-model-of-cln2-neuronal-ceroid-lipofuscinosis-after-intracerebroventricular-gene-therapy-that-delays-neurological-disease-progression
#19
M L Katz, G C Johnson, S B Leach, B G Williamson, J R Coates, R E H Whiting, D P Vansteenkiste, M S Whitney
CLN2 neuronal ceroid lipofuscinosis is a hereditary lysosomal storage disease with primarily neurological signs that results from mutations in TPP1, which encodes the lysosomal enzyme tripeptidyl peptidase-1 (TPP1). Studies using a canine model for this disorder demonstrated that delivery of TPP1 enzyme to the cerebrospinal fluid (CSF) by intracerebroventricular administration of an AAV-TPP1 vector resulted in substantial delays in the onset and progression of neurological signs and prolongation of life span...
April 2017: Gene Therapy
https://www.readbyqxmd.com/read/28075429/telomerase-specific-oncolytic-adenovirus-expressing-trail-suppresses-peritoneal-dissemination-of-gastric-cancer
#20
W Zhou, S Dai, H Zhu, Z Song, Y Cai, J B Lee, Z Li, X Hu, B Fang, C He, X Huang
Peritoneal dissemination is the most common condition of metastasis in gastric cancer. The survival duration of a patient with advanced stage gastric cancer, may be improved by gene therapy. In this study, we used an oncolytic adenovirus vector (Ad/TRAIL-E1) that expresses both the TRAIL and E1A genes under the control of a tumor-specific promoter. We evaluated the anti-tumor effect of Ad/TRAIL-E1 on gastric cancer cells in vitro, as well as in vivo in a xenograft peritoneal carcinomatosis mouse model. Our data showed that Ad/TRAIL-E1 induced TRAIL-mediated apoptosis in gastric cancer cell lines, but not in the normal cell lines...
April 2017: Gene Therapy
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