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Gene Therapy

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https://www.readbyqxmd.com/read/28905887/an-efficient-non-viral-dendritic-vector-for-gene-delivery-in-tissue-engineering
#1
D P Walsh, A Heise, F J O'Brien, S-A Cryan
Recent developments within the field of tissue engineering (TE) have shown that biomaterial scaffold systems can be augmented via the incorporation of gene therapeutics. The objective of this study was to assess the potential of the activated polyamidoamine dendrimer (dPAMAM) transfection reagent (Superfect(TM)) as a gene delivery system to mesenchymal stem cells (MSCs) in both monolayer and 3D culture on collagen based scaffolds. dPAMAM-pDNA polyplexes at a mass ratio (M:R) 10:1 (dPAMAM : pDNA) (1 ug pDNA) were capable of facilitating prolonged reporter gene expression in monolayer MSCs which was superior to that facilitated using polyethylenimine (PEI)-pDNA polyplexes (2 ug pDNA)...
September 14, 2017: Gene Therapy
https://www.readbyqxmd.com/read/28905886/time-series-oligonucleotide-count-to-assign-antiviral-sirnas-with-long-utility-fit-in-the-big-data-era
#2
K Wada, Y Wada, Y Iwasaki, T Ikemura
Oligonucleotides are key elements of nucleic acid therapeutics such as small interfering RNAs (siRNAs). Influenza and Ebolaviruses are zoonotic RNA viruses mutating very rapidly, and their sequence changes must be characterized intensively to design therapeutic oligonucleotides with long utility. Focusing on a total of 182 experimentally validated siRNAs for influenza A, B and Ebolaviruses compiled by the siRNA database, we conducted time-series analyses of occurrences of siRNA targets in these viral genomes...
September 14, 2017: Gene Therapy
https://www.readbyqxmd.com/read/28905885/long-term-correction-of-hemophilia-a-mice-following-lentiviral-mediated-delivery-of-an-optimized-canine-factor-viii-gene
#3
J M Staber, M J Pollpeter, C-G Anderson, M Burrascano, A L Cooney, P L Sinn, D T Rutkowski, W C Raschke, P B McCray
Current therapies for hemophilia A include frequent prophylactic or on-demand intravenous factor treatments which are costly, inconvenient and may lead to inhibitor formation. Viral vector delivery of factor VIII (FVIII) cDNA has the potential to alleviate the debilitating clotting defects. Lentiviral-based vectors delivered to murine models of hemophilia A mediate phenotypic correction. However, a limitation of lentiviral-mediated FVIII delivery is inefficient transduction of target cells. Here, we engineer a feline immunodeficiency virus (FIV) -based lentiviral vector pseudotyped with the baculovirus GP64 envelope glycoprotein to mediate efficient gene transfer to mouse hepatocytes...
September 14, 2017: Gene Therapy
https://www.readbyqxmd.com/read/28880021/long-term-expression-of-melanopsin-and-channelrhodopsin-causes-no-gross-alterations-in-the-dystrophic-dog-retina
#4
B Ameline, K-T Tshilenge, M Weber, M Biget, L Libeau, R Caplette, A Mendes-Madeira, N Provost, C Guihal, S Picaud, P Moullier, V Pichard, T Cronin, C Isiegas
Several preclinical studies have investigated the potential of algal channelrhodopsin and human melanopsin as optogenetic tools for vision restoration. In the present study, we assessed the potentially deleterious effects of long-term expression of these optogenes on the diseased retina in a large animal model of retinal degeneration, the RPE65-deficient Briard dog model of Leber congenital amaurosis. Intravitreal injection of adeno-associated virus vectors expressing channelrhodopsin and melanopsin had no effect on retinal thickness over a 16-month period post injection...
September 7, 2017: Gene Therapy
https://www.readbyqxmd.com/read/28880020/novel-gp64-envelope-variants-for-improved-delivery-to-human-airway-epithelial-cells
#5
P L Sinn, B-Y Hwang, N Li, J L S Ortiz, E Shirazi, K R Parekh, A L Cooney, D V Schaffer, P B McCray
Lentiviral vectors pseudotyped with the baculovirus envelope protein GP64 transduce primary cultures of human airway epithelia (HAE) at their apical surface. Our goal in this study was to harness a directed evolution approach to develop a novel envelope glycoprotein with increased transduction properties for HAE. Using error-prone PCR, a library of GP64 mutants was generated and used to prepare a diverse pool of lentiviral virions pseudotyped with GP64 variants. The library was serially passaged on HAE and three GP64 mutations were recovered...
September 7, 2017: Gene Therapy
https://www.readbyqxmd.com/read/28880019/market-access-of-spinraza-nusinersen-for-spinal-muscular-atrophy-intellectual-property-rights-pricing-value-and-coverage-considerations
#6
S Simoens, I Huys
No abstract text is available yet for this article.
September 7, 2017: Gene Therapy
https://www.readbyqxmd.com/read/28880018/spinraza-the-patient-perspective
#7
S Madipalli
No abstract text is available yet for this article.
September 7, 2017: Gene Therapy
https://www.readbyqxmd.com/read/28880017/will-rising-prices-and-budget-constraints-prevent-patients-from-accessing-novel-gene-therapies
#8
A Kent, J Spink
No abstract text is available yet for this article.
September 7, 2017: Gene Therapy
https://www.readbyqxmd.com/read/28872643/tropism-of-engineered-and-evolved-recombinant-aav-serotypes-in-the-rd1-mouse-and-ex-vivo-primate-retina
#9
D G Hickey, T L Edwards, A R Barnard, M S Singh, S R de Silva, M E McClements, J G Flannery, M W Hankins, R E MacLaren
There is much debate on the adeno-associated virus (AAV) serotype that best targets specific retinal cell types and the route of surgical delivery-intravitreal or subretinal. This study compared three of the most efficacious AAV vectors known to date in a mouse model of retinal degeneration (rd1 mouse) and macaque and human retinal explants. GFP driven by a ubiquitous promoter was packaged into three AAV capsids: AAV2/8(Y733F), AAV2/2(quad Y-F) and AAV2/2(7m8). Overall AAV2/2(7m8) transduced the largest area of retina and resulted in the highest level of GFP expression, followed by AAV2/2(quad Y-F) and AAV2/8(Y733F)...
September 5, 2017: Gene Therapy
https://www.readbyqxmd.com/read/28853717/in-utero-delivery-of-raav2-9-induces-neuronal-expression-of-the-transgene-in-the-brain-towards-new-models-of-parkinson-s-disease
#10
L Chansel-Debordeaux, M Bourdenx, S Dovero, V Grouthier, N Dutheil, A Espana, L Groc, C Jimenez, E Bezard, B Dehay
Animal models are essential tools for basic pathophysiological research as well as validation of therapeutic strategies for curing human diseases. However, technical difficulties associated with classical transgenesis approaches in rodent species higher than mus musculus have prevented this long-awaited development. The availability of viral-mediated gene delivery systems in the past few years has stimulated the production of viruses with unique characteristics. For example, the recombinant adeno-associated virus serotype 9 (rAAV2/9) crosses the blood-brain barrier, is capable of transducing developing cells and neurons after intravenous injection and mediates long-term transduction...
August 30, 2017: Gene Therapy
https://www.readbyqxmd.com/read/28846076/a-new-combination-therapy-for-asthma-using-dual-function-dexamethsone-conjugated-polyethylenimine-and-vitamin-d-binding-protein-sirna
#11
M Choi, J Gu, M Lee, T Rhim
Asthma is a multifactorial disease that is influenced by the interaction of genetic and environmental factors. Due to its complex nature, there is no cure for asthma currently. Instead, reliever and controller medications are used to treat asthma. Unfortunately, conventional treatments do not work in some severe cases of asthma. In addition, there may be adverse, systemic effects of long-term treatment with high-dose inhaled corticosteroids as a controller medication. Therefore, we attempted to develop a novel combination therapy for asthma...
August 28, 2017: Gene Therapy
https://www.readbyqxmd.com/read/28832561/stent-based-delivery-of-adeno-associated-viral-vectors-with-sustained-vascular-transduction-and-inos-mediated-inhibition-of-in-stent-restenosis
#12
I Fishbein, D T Guerrero, I S Alferiev, J B Foster, N G Minutolo, M Chorny, A M Monteys, K H Driesbaugh, C Nagaswami, R J Levy
In-stent restenosis remains an important clinical problem in the era of drug eluting stents. Development of clinical gene therapy protocols for the prevention and treatment of in-stent restenosis is hampered by the lack of adequate local delivery systems. Herein we describe a novel stent-based gene delivery platform capable of providing local arterial gene transfer with adeno-associated viral (AAV) vectors. This system exploits the natural affinity of protein G (PrG) to bind to the Fc region of mammalian IgG, making PrG a universal adaptor for surface immobilization of vector-capturing antibodies (Ab)...
August 23, 2017: Gene Therapy
https://www.readbyqxmd.com/read/28820502/oligonucleotides-targeting-periostin-ameliorates-pulmonary-fibrosis
#13
A Tomaru, T Kobayashi, J A Hinneh, P B Tonto, C N D' Alessandro-Gabazza, H Fujimoto, K Fujiwara, Y Takahashi, M Ohnishi, T Yasuma, K Nishihama, M Yoshino, K Takao, M Toda, T Totoki, Y Takei, K Yoshikawa, O Taguchi, E C Gabazza
Idiopathic pulmonary fibrosis (IPF) is a fatal disease with a median survival of 3 to 4 years after diagnosis. It is the most frequent form of a group of interstitial pneumonias of unknown etiology. Current available therapies prevent deterioration of lung function but no therapy has shown to improve survival. Periostin is a matricellular protein of the fasciclin 1 family. There is increased deposition of periostin in lung fibrotic tissues. Here, we evaluated whether small interfering RNA or antisense oligonucleotide against periostin inhibit lung fibrosis by direct administration into the lung by intranasal route...
August 18, 2017: Gene Therapy
https://www.readbyqxmd.com/read/28820501/interleukin-armed-chimeric-antigen-receptor-modified-t-cells-for-cancer-immunotherapy
#14
REVIEW
Y Huang, D Li, D-Y Qin, H-F Gou, W Wei, Y-S Wang, Y-Q Wei, W Wang
Chimeric antigen receptor-modified T cells (CAR-T) are endowed with cytotoxic specificity to tumor cells. Although CAR-T-based cancer immunotherapy presents curable therapeutic potential for hematological malignancies, achieving substantial efficacy for solid tumors remain challenging. Researchers have exploited many strategies to enhance the anti-tumor efficacy of CAR-T cells for solid tumors, among which cytokine-armed CAR-T cells improve the proliferation, survival, homing and other properties of CAR-T cells...
August 18, 2017: Gene Therapy
https://www.readbyqxmd.com/read/28805798/repeated-aav-mediated-gene-transfer-by-serotype-switching-enables-long-lasting-therapeutic-levels-of-hugt1a1-enzyme-in-a-mouse-model-of-crigler-najjar-syndrome-type-i
#15
L Bočkor, G Bortolussi, A Iaconcig, G Chiaruttini, C Tiribelli, M Giacca, F Benvenuti, L Zentilin, A F Muro
Adeno-associated virus (AAV)-mediated gene therapy is a promising strategy to treat liver-based monogenic diseases. However, two major obstacles limit its success: first, vector dilution in actively dividing cells, such as hepatocytes in neonates/children, due to the non-integrating nature of the vector; second, development of an immune response against the transgene and/or viral vector. Crigler-Najjar Syndrome Type I (CNSI) is a rare monogenic disease with neonatal onset, caused by mutations in the liver-specific UGT1 gene, with toxic accumulation of unconjugated bilirubin in plasma, tissues and brain...
August 14, 2017: Gene Therapy
https://www.readbyqxmd.com/read/28805797/human-genomics-projects-and-precision-medicine
#16
REVIEW
F Carrasco-Ramiro, R Peiró-Pastor, B Aguado
The completion of the Human Genome Project (HGP) in 2001 opened the floodgates to a deeper understanding of medicine. There are dozens of HGP-like projects which involve from a few tens to several million genomes currently in progress, which vary from having specialised goals or a more general approach. However, data generation, storage, management and analysis in public and private cloud computing platforms have raised concerns about privacy and security. The knowledge gained from further research has changed the field of genomics and is now slowly permeating into clinical medicine...
August 14, 2017: Gene Therapy
https://www.readbyqxmd.com/read/28805796/bifidobacterium-breve-as-a-delivery-vector-of-il-24-gene-therapy-for-head-and-neck-squamous-cell-carcinoma-in-vivo
#17
L Wang, I Vuletic, D Deng, W Crielaard, Z Xie, K Zhou, J Zhang, H Sun, Q Ren, C Guo
Beneficial bacteria are becoming ever more popular gene delivery method for hypoxia- tumor targeting in vivo. In this study we investigated the therapeutic effect of new recombinant Bifidobacterium breve strain expressing IL-24 gene (B.breve-IL24) on head and neck tumor xenograft in mice. Briefly, B.breve transformants were obtained through electro-transformation. Bacteria-tumor targeting ability were analyzed in vivo over different time points (1, 3 and 7 days post- bacteria injection). Furthermore, the therapeutic effect of bacteria on tumor cells in vivo were analyzed as follows: 30 Balb/c nude mice bearing subcutaneous tumor were randomly divided in three groups (Drug group, GFP group and Saline group)...
August 14, 2017: Gene Therapy
https://www.readbyqxmd.com/read/28771235/systemic-gene-delivery-transduces-the-enteric-nervous-system-of-guinea-pigs-and-cynomolgus-macaques
#18
S E Gombash, C J Cowley, J A Fitzgerald, C A Lepak, M G Neides, K Hook, L J Todd, G-D Wang, C Mueller, B K Kaspar, E C Bielefeld, A J Fischer, J D Wood, K D Foust
Characterization of adeno-associated viral vector (AAV) mediated gene delivery to the enteric nervous system (ENS) was recently described in mice and rats. In these proof-of-concept experiments, we show that intravenous injections of clinically relevant AAVs can transduce the ENS in guinea pigs and non-human primates. Neonatal guinea pigs were given intravenous injections of either AAV8 or AAV9 vectors that contained a green fluorescent protein (GFP) expression cassette or PBS. Piglets were euthanized three weeks post-injection and tissues were harvested for immunofluorescent analysis...
August 3, 2017: Gene Therapy
https://www.readbyqxmd.com/read/28771234/aav5-mihtt-gene-therapy-demonstrates-suppression-of-mutant-huntingtin-aggregation-and-neuronal-dysfunction-in-a-rat-model-of-huntington-s-disease
#19
J Miniarikova, V Zimmer, R Martier, C C Brouwers, C Pythoud, K Richetin, M Rey, J Lubelski, M M Evers, S J van Deventer, H Petry, N Déglon, P Konstantinova
Huntington's disease (HD) is a fatal progressive neurodegenerative disease caused by a mutation in the huntingtin (HTT) gene. To date, there is no treatment to halt or reverse the course of HD. Lowering of either total or only the mutant HTT expression is expected to have therapeutic benefit. This can be achieved by engineered micro (mi)RNAs targeting HTT transcripts and delivered by an adeno-associated viral (AAV) vector. We have previously showed a miHTT construct to induce total HTT knock-down in Hu128/21 HD mice, while miSNP50T and miSNP67T constructs induced allele-selective HTT knock-down in vitro...
August 3, 2017: Gene Therapy
https://www.readbyqxmd.com/read/28771233/impact-of-germline-and-somatic-brca1-2-mutations-tumor-spectrum-and-detection-platforms
#20
REVIEW
H Wu, X Wu, Z Liang
The BRCA1/2 genes are long and complex and mutation carriers are at risk of developing malignancies, mainly of gynecological origin. Various mutations arise in these genes and their characterization is a time consuming, cost intensive, complicated process. Tumors of BRCA1/2 origin have distinct molecular and histological features that can impact responses to therapy. Therefore, detection of these mutations constitutes an important step in the risk assessment, prevention strategy and treatment of subjects. Although Sanger sequencing is the gold standard for the detection of genetic mutations, several next generation sequencing-based high throughput platforms have been developed and adapted for the detection of BRCA1/2 mutations...
August 3, 2017: Gene Therapy
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