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Gene Therapy

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https://www.readbyqxmd.com/read/28540936/antigen-presenting-cell-targeted-lentiviral-vectors-do-not-support-the-development-of-productive-t-cell-effector-responses-implications-for-in-vivo-targeted-vaccine-delivery
#1
C Goyvaerts, Y De Vlaeminck, D Escors, S Lienenklaus, M Keyaerts, G Raes, K Breckpot
Targeting transgene expression specifically to antigen-presenting cells (APCs) has been put forward as a promising strategy to direct the immune system towards immunity. We developed the nanobody-display technology to restrict the tropism of lentiviral vectors (LVs) to APCs. However, we observed that immunization with APC-targeted LVs (DC2.1-LVs) did not evoke strong antigen-specific T-cell immunity when compared to immunization with broad tropism LVs (VSV.G-LVs). In this study, we report that VSV.G-LVs are more immunogenic than DC2...
May 25, 2017: Gene Therapy
https://www.readbyqxmd.com/read/28530652/cd34-cells-from-dental-pulp-stem-cells-with-a-zfn-mediated-and-homology-driven-repair-mediated-locus-specific-knock-in-of-an-artificial-%C3%AE-globin-gene
#2
S Chattong, O Ruangwattanasuk, W Yindeedej, A Setpakdee, K Manotham
In humans, mutations in the β-globin gene (HBB) have two important clinical manifestations: β-thalassemia and sickle cell disease. The progress in genome editing and stem cell research may be relevant to the treatment of β-globin-related diseases. In this work, we employed zinc finger nuclease (ZFN)-mediated gene integration of synthetic β-globin cDNA into HBB loci, thus correcting almost all β-globin mutations. The integration was achieved in both HEK 293 cells and isolated dental pulp stem cell (DPSCs)...
May 22, 2017: Gene Therapy
https://www.readbyqxmd.com/read/28504658/cure-sma-and-our-patient-community-celebrate-the-first-approved-drug-for-sma
#3
REVIEW
J Glascock, M Lenz, K Hobby, J Jarecki
Cure SMA is dedicated to the treatment and cure of spinal muscular atrophy (SMA), the number one genetic cause of death for infants. We fund groundbreaking research and provide families the support they need for today. Today, we have more than 115 000 members and supporters, with 35 volunteer chapters throughout the United States. In the last year, Cure SMA provided direct services to several thousand families. Since 1984, we've led and invested in the research that has made today's breakthroughs possible. With deep connections and expertise in both the patient and research communities, we have been uniquely positioned to direct funds to where they can make the greatest difference as quickly as possible...
May 15, 2017: Gene Therapy
https://www.readbyqxmd.com/read/28504657/engineering-liposomal-nanoparticles-for-targeted-gene-therapy
#4
REVIEW
C Zylberberg, K Gaskill, S Pasley, S Matosevic
Recent mechanistic studies have attempted to deepen our understanding of the process by which liposome-mediated delivery of genetic material occurs. Understanding the interactions between lipid nanoparticles and cells is still largely elusive. Liposome-mediated delivery of genetic material faces systemic obstacles alongside entry into the cell, endosomal escape, lysosomal degradation and nuclear uptake. Rational design approaches for targeted delivery have been developed to reduce off-target effects and enhance transfection...
May 15, 2017: Gene Therapy
https://www.readbyqxmd.com/read/28504656/effect-of-il-1%C3%AE-tnf-%C3%AE-and-igf-1-on-trans-endothelial-passage-of-synthetic-vectors-through-an-in-vitro-vascular-endothelial-barrier-of-striated-muscle
#5
J-P Gomez, C Gonçalves, C Pichon, P Midoux
When administrated in the blood circulation, plasmid DNA (pDNA) complexed with synthetic vectors must pass through a vascular endothelium to transfect underlying tissues. Under inflammatory condition, cytokines effects can modify the endothelium integrity. Here, the trans-endothelial passage (TEP) of DNA complexes including polyplexes, lipoplexes and lipopolyplexes was investigated in the presence of TNF-α-, IL-1β or IGF-1. The experiments were performed by using an in vitro model comprising a monolayer of mouse cardiac endothelial cells (MCEC) seeded on a trans-well insert and the transfection of C2C12 myoblasts cultured on the lower chamber as read out of TEP...
May 15, 2017: Gene Therapy
https://www.readbyqxmd.com/read/28492521/coinjection-of-il2-dna-enhances-e7-specific-antitumor-immunity-elicited-by-intravaginal-therapeutic-hpv-dna-vaccination-with-electroporation
#6
Y Sun, S Peng, A Yang, E Farmer, T-C Wu, Chien-Fu Hung
The generation and use of therapeutic human papillomavirus (HPV) DNA vaccines represent an appealing treatment method against HPV-associated cervical cancer due to their safety and durability. Previously, we created a therapeutic HPV DNA vaccine candidate by linking the HPV16-E7 DNA sequence to calreticulin (CRT/E7), which we showed could generate significant E7-specific cytotoxic T lymphocyte (CTL)-mediated anti-tumor immune responses against HPV16 oncogenes expressing murine tumor model TC-1. Here we assess the therapeutic efficacy of intravaginal immunization with pcDNA3-CRT/E7 followed by electroporation...
May 11, 2017: Gene Therapy
https://www.readbyqxmd.com/read/28467403/building-the-patient-community
#7
REVIEW
F Raffai, O Timmis
There are many challenges in conducting rare disease research. The conditions are often poorly understood, small patient populations are dispersed around the world, and there are limited funding opportunities. Patient groups can serve as a key partner in overcoming these challenges, as they understand the impact of rare conditions on patients' lives. This gives patient groups valuable scientific insights into the disease. This can be used to create research strategies, address research bottlenecks and directly fund research that appropriately addresses patient needs...
May 11, 2017: Gene Therapy
https://www.readbyqxmd.com/read/28467402/new-treatments-for-serious-conditions-ethical-implications
#8
REVIEW
N M P King, C E Bishop
Approval of Spinraza (nusinersen) for treatment of spinal muscular atrophy prompts consideration of a number of ethical issues that arise whenever a new treatment is proposed for a serious condition, especially one that is rare and can devastatingly affect children. Patients, families, clinicians, researchers, institutions and policymakers all must take account of the ways that newly available treatments affect informed and shared decision-making about therapeutic and research options. The issues to consider include: addressing what is still uncertain and unknown; the possibility that potential benefits will be exaggerated and potential harms underemphasized in the media, by advocacy organizations, and in consent forms and processes; the high cost of many novel drugs and biologics; the effects of including conditions of variable phenotype in state-mandated newborn screening panels; and how new treatments can change the standard of care, altering what is and is not known about a disorder and posing challenges for decision-making at both individual and policy levels...
May 11, 2017: Gene Therapy
https://www.readbyqxmd.com/read/28485723/developing-gene-and-cell-therapies-for-rare-diseases-an-opportunity-for-synergy-between-academia-and-industry
#9
REVIEW
F Mavilio
For the last twenty years, academic research has been the major, and often only, driving force behind the spectacular development of gene transfer technology for the therapy of rare genetic diseases. Investors and industry became eventually interested in gene and cell therapy, due to the success of a series of pioneering clinical trials that proved efficacy and safety of last-generation technology, and to favorable orphan drug legislation in both Europe and the United States. Developing this forms of therapy is however complex and requires skills and knowledge not necessary available to the industry, which is better placed to develop processes and products and put them on the market...
May 9, 2017: Gene Therapy
https://www.readbyqxmd.com/read/28485722/how-the-discovery-of-iss-n1-led-to-the-first-medical-therapy-for-spinal-muscular-atrophy
#10
REVIEW
N N Singh, M D Howell, E J Androphy, R N Singh
Spinal muscular atrophy (SMA), a prominent genetic disease of infant mortality, is caused by low levels of survival motor neuron (SMN) protein owing to deletions or mutations of the SMN1 gene. SMN2, a nearly identical copy of SMN1 present in humans, cannot compensate for the loss of SMN1 due to predominant skipping of exon 7 during pre-mRNA splicing. With the recent FDA approval of nusinersen (Spinraza™), the potential for correction of SMN2 exon 7 splicing as a SMA therapy has been affirmed. Nusinersen is an antisense oligonucleotide that targets intronic splicing silencer N1 (ISS-N1) discovered in 2004 at the University of Massachusetts Medical School...
May 9, 2017: Gene Therapy
https://www.readbyqxmd.com/read/28485721/sost-silencing-promotes-proliferation-and-invasion-and-reduces-apoptosis-of-retinoblastoma-cells-by-activating-wnt-%C3%AE-catenin-signaling-pathway
#11
T Wu, L-N Wang, D-R Tang, F-Y Sun
This study aimed to investigate the effects of SOST and the Wnt/β-catenin signaling pathway on the proliferation, migration, invasion, and apoptosis of human retinoblastoma cells. Fifty-five retinoblastoma and 21 normal retinal tissue samples were collected as the case group and control group, respectively. HXO-RB44 and SO-RB50 cells were selected and assigned into blank, negative control (NC), siRNA1, siRNA2, siRNA3, IWR-1-endo 1, IWR-1-endo 2, and IWR-1-endo 3 groups. Quantitative real-time polymerase chain reaction (qRT-PCR) was applied to detect the expression of SOST, Wnt1, and β-catenin in the collected tissue samples...
May 9, 2017: Gene Therapy
https://www.readbyqxmd.com/read/28485720/herpesvirus-micrornas-for-use-in-gene-therapy-immune-evasion-strategies
#12
REVIEW
S T F Bots, R C Hoeben
Transplantation of allogeneic cells as well as of genetically corrected autologous cells are potent approaches to restore cellular functions in patients suffering from genetic diseases. The recipient's immune responses against non-self-antigens may compromise the survival of the grafted cells. Recipients of the graft may therefore require life-long treatment with immunosuppressive drugs. An alternative approach to reduce graft rejection could involve the use of immune-evasion molecules. Expression of such molecules in cells of the graft may subvert recognition by the host's immune system...
May 9, 2017: Gene Therapy
https://www.readbyqxmd.com/read/28471431/current-application-of-crispr-cas9-gene-editing-technique-to-eradication-of-hiv-aids
#13
REVIEW
Z Huang, A Tomitaka, A Raymond, M Nair
Human immunodeficiency virus (HIV)/acquired immunodeficiency syndrome (AIDS) remains a major health hazard despite significant advances in prevention and treatment of HIV infection. The major reason for the persistence of HIV/AIDS is the inability of existing treatments to clear or eradicate the multiple HIV reservoirs that exist in the human body. To suppress the virus replication and rebound, HIV/AIDS patients must take life-long antiviral medications. The clustered regularly interspaced palindromic repeats (CRISPR)/CRISPR-associated nuclease 9 (Cas9) system is an emerging gene-editing technique with the potential to eliminate or disrupt HIV-integrated genomes or HIV-infected cells from multiple HIV reservoirs, which could result in the complete cure of HIV/AIDS...
May 4, 2017: Gene Therapy
https://www.readbyqxmd.com/read/28440798/improved-gene-delivery-to-adult-mouse-spinal-cord-through-the-use-of-engineered-hybrid-adeno-associated-viral-serotypes
#14
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...
May 4, 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
#15
M L Palma, P Duangkhae, B Douradinha, I F T Viana, P O Rigato, R Dhalia, R B Mailliard, S M Barratt-Boyes, E J M Nascimento, T M Oshiro, A J da Silva Duarte, E T A Marques
Class II transactivator (CIITA) induces transcription of major histocompatibility complex (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...
May 4, 2017: Gene Therapy
https://www.readbyqxmd.com/read/28387759/characterization-of-retroviral-infectivity-and-superinfection-resistance-during-retrovirus-mediated-transduction-of-mammalian-cells
#16
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 (RV)-based libraries, which takes advantage of retroviral superinfection resistance. However, the dynamic features of RV-mediated transduction are not well characterized. Here, we engineered two murine stem cell virus-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...
May 4, 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
#17
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
#18
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
#19
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
#20
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...
May 2017: Gene Therapy
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