keyword
MENU ▼
Read by QxMD icon Read
search

gene delivery

keyword
https://www.readbyqxmd.com/read/29792920/inner-ear-drug-delivery-recent-advances-challenges-and-perspective
#1
Jinsong Hao, S Kevin Li
Effective and safe treatment of auditory and vestibular diseases has become increasingly dependent on inner ear drug delivery systems. This review highlights recent advances in inner ear drug delivery research and technologies. The focus is on strategies to overcome delivery barriers and to improve drug residence and targeting, with special attention to in vivo animal and human studies. The research in gene and stem cell delivery to the inner ear is briefly reviewed. Newly developed research tools to address experimental challenges and safety issues are discussed...
May 21, 2018: European Journal of Pharmaceutical Sciences
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
#2
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/29792369/gene-delivery-in-the-cornea-in-vitro-ex-vivo-evaluation-of-solid-lipid-nanoparticle-based-vectors
#3
Mónica Vicente-Pascual, Andrea Albano, María Á Solinís, Loredana Serpe, Alicia Rodríguez-Gascón, Federica Foglietta, Elisabetta Muntoni, Josune Torrecilla, Ana Del Pozo-Rodríguez, Luigi Battaglia
AIM: Inflammation is a process that underlies sight-threatening ocular surface diseases, and gene supplementation with the plasmid that encodes for p-IL10 will allow the sustained de novo synthesis of the cytokine to occur in corneal cells, and provide a long-term anti-inflammatory effect. This work describes the development of solid lipid nanoparticle systems for the delivery of p-IL10 to transfect the cornea. RESULTS: In vitro, vectors showed suitable features as nonviral vectors (size, ζ-potential, DNA binding, protection and release), and they were able to enter and transfect human corneal epithelial cells...
May 24, 2018: Nanomedicine
https://www.readbyqxmd.com/read/29792157/long-term-health-and-germline-transmission-in-transgenic-cattle-following-transposon-mediated-gene-transfer
#4
Soo-Young Yum, Song-Jeon Lee, Sin-Gi Park, In-Gang Shin, Sang-Eun Hahn, Woo-Jae Choi, Hee-Soo Kim, Hyeong-Jong Kim, Seong-Hun Bae, Je-Hyeong Lee, Joo-Yeong Moon, Woo-Sung Lee, Ji-Hyun Lee, Choong-Il Lee, Seong-Jin Kim, Goo Jang
BACKGROUND: Transposon-mediated, non-viral gene delivery is a powerful tool for generating stable cell lines and transgenic animals. However, as multi-copy insertion is the preferred integration pattern, there is the potential for uncontrolled changes in endogenous gene expression and detrimental effects in cells or animals. Our group has previously reported on the generation of several transgenic cattle by using microinjection of the Sleeping Beauty (SB) and PiggyBac (PB) transposons and seeks to explore the long-term effects of this technology on cattle...
May 23, 2018: BMC Genomics
https://www.readbyqxmd.com/read/29791775/vaginal-microbiome-in-early-pregnancy-and-subsequent-risk-of-spontaneous-preterm-birth-a-case-control-study
#5
Negar Tabatabaei, A Murat Eren, Luis B Barreiro, Vania Yotova, Anne Dumaine, Catherine Allard, William D Fraser
OBJECTIVES: To explore differences in vaginal microbiome between preterm and term deliveries. DESIGN: Nested case-control study in 3D cohort. SETTING: Quebec, Canada. SAMPLE: Ninety-four cases with spontaneous preterm birth (17 early (<34 wk) and 77 late (34-36 wk) preterm birth) and 356 controls with term delivery (≥37 wk). METHODS: To assess vaginal microbiome by sequencing the V4 region of 16S ribosomal-RNA (rRNA) gene in swabs self-collected early pregnancy...
May 23, 2018: BJOG: An International Journal of Obstetrics and Gynaecology
https://www.readbyqxmd.com/read/29790803/ultrasound-guided-delivery-of-thymidine-kinase-nitroreductase-dual-therapeutic-genes-by-pegylated-plga-pie-nanoparticles-for-enhanced-triple-negative-breast-cancer-therapy
#6
Rammohan Devulapally, Taehwa Lee, Aarohi Barghava-Shah, Thillai V Sekar, Kira Foygel, Sunitha V Bachawal, Jürgen K Willmann, Ramasamy Paulmurugan
AIM: Triple-negative breast cancer (TNBC) is an aggressive breast cancer subtype. Since no targeted therapy is available, gene-directed enzyme prodrug therapy (GDEPT) could be an attractive strategy for treating TNBC. MATERIALS & METHODS:  Polyethylene glycol (PEG)ylated-poly(lactic-co-glycolic acid)/polyethyleneimine nanoparticles (PLGA/PEI NPs) were synthesized and complexed with TK-NTR fusion gene. Ultrasound (US) and microbubble (MB) mediated sonoporation was used for efficient delivery of the TK-NTR-DNA-NP complex to TNBC tumor in vivo for cancer therapy...
May 2018: Nanomedicine
https://www.readbyqxmd.com/read/29790082/chronic-administration-of-pimozide-fails-to-attenuate-motor-and-pathological-deficits-in-two-mouse-models-of-amyotrophic-lateral-sclerosis
#7
Silvia Pozzi, Sai Sampath Thammisetty, Jean-Pierre Julien
Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease which presently does not have any efficient therapeutic approach. Pimozide, a Food and Drug Administration (FDA)-approved neuroepileptic drug, has been recently proposed as a promising treatment for ALS patients based on apparent stabilization of right hand muscles after a short-time administration. A new clinical trial started at the end of 2017 to recruit patients with a prolonged drug delivery schedule. Here, our aim was to investigate the effects of chronic administration of pimozide on disease progression and pathological events in two mouse models of ALS...
May 22, 2018: Neurotherapeutics: the Journal of the American Society for Experimental NeuroTherapeutics
https://www.readbyqxmd.com/read/29789683/bmp-2-restoration-aids-in-recovery-from-liver-fibrosis-by-attenuating-tgf-%C3%AE-1-signaling
#8
Yueh-Hua Chung, Ying-Hsien Huang, Tien-Huei Chu, Chun-Lin Chen, Pey-Ru Lin, Shih-Chung Huang, Deng-Chyang Wu, Chao-Cheng Huang, Tsung-Hui Hu, Ying-Hsien Kao, Ming-Hong Tai
Transforming growth factor-β (TGF-β) plays a central role in hepatic fibrogenesis. This study investigated the function and mechanism of bone morphogenetic protein-2 (BMP-2) in regulation of hepatic fibrogenesis. BMP-2 expression in fibrotic liver was measured in human tissue microarray and mouse models of liver fibrosis induced by bile duct ligation surgery or carbon tetrachloride administration. Adenovirus-mediated BMP-2 gene delivery was used to test the prophylactic effect on liver fibrosis. Primary hepatic stellate cells (HSC), HSC-T6 and clone-9 cell lines were used to study the interplay between BMP-2 and TGF-β1...
May 22, 2018: Laboratory Investigation; a Journal of Technical Methods and Pathology
https://www.readbyqxmd.com/read/29788394/rna-interference-of-the-muscle-actin-gene-in-bed-bugs-exploring-injection-versus-topical-application-for-dsrna-delivery
#9
Sanjay Basnet, Shripat T Kamble
Bed bugs are one the most troublesome household pests that feed primarily on human blood. RNA interference (RNAi) is currently being pursued as a potential tool for insect population management and has shown efficacy against some phytophagous insects. We evaluated the different techniques to deliver dsRNA specific to bed bug muscle actin (dsactin) into bed bugs. Initially, stability of dsRNA in human blood was studied to evaluate the feasibility of feeding method. Adult bed bugs were injected with dsRNA between last thoracic segment and first abdominal segment on the ventral side, with a dose of 0...
May 1, 2018: Journal of Insect Science
https://www.readbyqxmd.com/read/29788236/a-novel-adeno-associated-virus-capsid-with-enhanced-neurotropism-corrects-a-lysosomal-transmembrane-enzyme-deficiency
#10
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/29788117/are-we-ready-to-improve-phosphorus-homeostasis-in-rice
#11
Stanislav Kopriva, Chengcai Chu
Phosphorus (P) is an essential macronutrient which often limits plant growth, but the phosphate rock used for fertilizer production is a finite resource. On the other hand, large amounts of P compounds are entering surface waters, leading to eutrophication. Therefore, improvement of phosphate use efficiency of crop plants is a major task for plant science. Rice as a staple crop has recently been a focus of such efforts with several major discoveries. New transporters controlling phosphate homeostasis in rice have been discovered...
May 17, 2018: Journal of Experimental Botany
https://www.readbyqxmd.com/read/29787950/macroporous-silica-nanoparticles-for-delivering-bcl2-function-converting-peptide-to-treat-multidrug-resistant-cancer-cells
#12
Weixia Xu, Pengjin Ge, Boning Niu, Xiaokun Zhang, Jie Liu, Jingjing Xie
The abundance of B cell lymphoma gene 2 (Bcl-2) is closely correlated with the resistance of cancer cells to chemotherapeutic agents, and a peptide derived from orphan nuclear receptor Nur77 can convert Bcl-2 from a protector to a killer of cancer cells. However, successful application of the Bcl-2-converting peptide to treat drug-resistant cancer cells depends on an efficient delivery carrier. Mesoporous silica nanoparticles (MSNs) have been extensively studied as promising candidates for small molecule drug delivery...
May 15, 2018: Journal of Colloid and Interface Science
https://www.readbyqxmd.com/read/29787895/poly-amidoamine-pamam-dendrimer-mediated-delivery-of-drug-and-pdna-sirna-for-cancer-therapy
#13
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/29787588/angiogenic-and-pleiotropic-effects-of-vegf165-and-hgf-combined-gene-therapy-in-a-rat-model-of-myocardial-infarction
#14
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/29786106/efficient-delivery-of-hbv-nls-sirnas-into-hepg2-2-15-cells-for-hbv-inhibition-through-novel-recombinant-pres1%C3%A2-tp-proteins
#15
Yanli Zeng, Zixi Li, Jia Shang, Yi Kang
Hepatitis B virus (HBV) infection and related liver complications remain severe public health problems worldwide. Previous investigations have shown that small interfering (si)RNAs can offer an effective strategy for the treatment of chronic hepatitis B. The present study aimed to develop a novel siRNA‑delivering system of therapeutic HBV nuclear localization sequence (NLS) siRNAs using the recombinant preS1‑truncated protamine (tP) proteins. The preS1 region of the LHB was used in place of scFv to construct the recombinant preS1‑tP proteins, which were applied to deliver siRNAs targeting the HBV NLS to inhibit HBV replication and infection in HepG2...
May 16, 2018: International Journal of Molecular Medicine
https://www.readbyqxmd.com/read/29785047/rationally-designed-aav2-and-aavrh8r-capsids-provide-improved-transduction-in-the-retina-and-brain
#16
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/29785035/optimized-delivery-of-sirna-into-3d-tumor-spheroid-cultures-in-situ
#17
R G Morgan, A C Chambers, D N Legge, S J Coles, A Greenhough, A C Williams
3D tissue culture provides a physiologically relevant and genetically tractable system for studying normal and malignant human tissues. Despite this, gene-silencing studies using siRNA has proved difficult. In this study, we have identified a cause for why traditional siRNA transfection techniques are ineffective in eliciting gene silencing in situ within 3D cultures and proposed a simple method for significantly enhancing siRNA entry into spheroids/organoids. In 2D cell culture, the efficiency of gene silencing is significantly reduced when siRNA complexes are prepared in the presence of serum...
May 21, 2018: Scientific Reports
https://www.readbyqxmd.com/read/29784932/molecular-size-modulates-pharmacokinetics-biodistribution-and-renal-deposition-of-the-drug-delivery-biopolymer-elastin-like-polypeptide
#18
Marija Kuna, Fakhri Mahdi, Alejandro R Chade, Gene L Bidwell
Elastin-like polypeptides (ELP) are engineered proteins that consist of repetitions of a five amino acid motif, and their composition is easily modified to adjust their physical properties and attach therapeutics. Because of the repetitive nature of the ELP sequence, polymer size is particularly amenable to manipulation. ELP fusion proteins are being actively developed as therapeutics for many disease applications, and how the ELP size and shape affects its pharmacokinetics and biodistribution is a critical question for the general field of ELP drug delivery...
May 21, 2018: Scientific Reports
https://www.readbyqxmd.com/read/29784586/ultrasound-mediated-gene-delivery-enhances-tendon-allograft-integration-in-mini-pig-ligament-reconstruction
#19
Maxim Bez, Thomas J Kremen, Wafa Tawackoli, Pablo Avalos, Dmitriy Sheyn, Galina Shapiro, Joseph C Giaconi, Shiran Ben David, Jess G Snedeker, Zulma Gazit, Katherine W Ferrara, Dan Gazit, Gadi Pelled
Ligament injuries occur frequently, substantially hindering routine daily activities and sports participation in patients. Surgical reconstruction using autogenous or allogeneic tissues is the gold standard treatment for ligament injuries. Although surgeons routinely perform ligament reconstructions, the integrity of these reconstructions largely depends on adequate biological healing of the interface between the ligament graft and the bone. We hypothesized that localized ultrasound-mediated, microbubble-enhanced therapeutic gene delivery to endogenous stem cells would lead to significantly improved ligament graft integration...
April 26, 2018: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/29783825/-clinical-and-molecular-analysis-of-two-chinese-siblings-with-bloom-syndrome
#20
M L Wu, X M Wang, J Li, Y Ding, Y Chen, G Y Chang, J Wang, Y P Shen
Objective: To expand the knowledge of the clinical and molecular characteristics of the children with Bloom syndrome. Methods: Clinical data of two siblings with classic Bloom syndrome of Shanghai Children's Medical Center from January 2009 to June 2017 were obtained and analyzed. The DNA of peripheral blood was collected from two Bloom syndrome siblings and their parents during 2015. The mutations were detected with high-throughput sequencing by Illumina sequencing platform. Results: The two siblings (probands) visited our department for short stature and growth retardation, they had full-term normal delivery after normal pregnancy of their mother...
May 2, 2018: Zhonghua Er Ke za Zhi. Chinese Journal of Pediatrics
keyword
keyword
89145
1
2
Fetch more papers »
Fetching more papers... Fetching...
Read by QxMD. Sign in or create an account to discover new knowledge that matter to you.
Remove bar
Read by QxMD icon Read
×

Search Tips

Use Boolean operators: AND/OR

diabetic AND foot
diabetes OR diabetic

Exclude a word using the 'minus' sign

Virchow -triad

Use Parentheses

water AND (cup OR glass)

Add an asterisk (*) at end of a word to include word stems

Neuro* will search for Neurology, Neuroscientist, Neurological, and so on

Use quotes to search for an exact phrase

"primary prevention of cancer"
(heart or cardiac or cardio*) AND arrest -"American Heart Association"