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https://www.readbyqxmd.com/read/28339457/targeted-aav5-smad7-gene-therapy-inhibits-corneal-scarring-in-vivo
#1
Suneel Gupta, Jason T Rodier, Ajay Sharma, Elizabeth A Giuliano, Prashant R Sinha, Nathan P Hesemann, Arkasubhra Ghosh, Rajiv R Mohan
Corneal scarring is due to aberrant activity of the transforming growth factor β (TGFβ) signaling pathway following traumatic, mechanical, infectious, or surgical injury. Altered TGFβ signaling cascade leads to downstream Smad (Suppressor of mothers against decapentaplegic) protein-mediated signaling events that regulate expression of extracellular matrix and myogenic proteins. These events lead to transdifferentiation of keratocytes into myofibroblasts through fibroblasts and often results in permanent corneal scarring...
2017: PloS One
https://www.readbyqxmd.com/read/28338823/transporters-involved-in-ph-and-k-homeostasis-affect-pollen-wall-formation-male-fertility-and-embryo-development
#2
Senthilkumar Padmanaban, Daniel D Czerny, Kara A Levin, Alexander R Leydon, Robert T Su, Timothy K Maugel, Yanjiao Zou, Salil Chanroj, Alice Y Cheung, Mark A Johnson, Heven Sze
Flowering plant genomes encode multiple cation/H+ exchangers (CHXs) whose functions are largely unknown. AtCHX17, AtCHX18, and AtCHX19 are membrane transporters that modulate K+ and pH homeostasis and are localized in the dynamic endomembrane system. Loss of function reduced seed set, but the particular phase(s) of reproduction affected was not determined. Pollen tube growth and ovule targeting of chx17chx18chx19 mutant pollen appeared normal, but reciprocal cross experiments indicate a largely male defect...
February 23, 2017: Journal of Experimental Botany
https://www.readbyqxmd.com/read/28337437/bioengineering-approaches-to-mature-human-pluripotent-stem-cell-derived-cardiomyocytes
#3
REVIEW
Xuetao Sun, Sara S Nunes
Human pluripotent stem cell-derived cardiomyocytes (hPSC-CM) represent a potential unlimited cell supply for cardiac tissue engineering and possibly regenerative medicine applications. However, hPSC-CMs produced by current protocols are not representative of native adult human cardiomyocytes as they display immature gene expression profile, structure and function. In order to improve hPSC-CM maturity and function, various approaches have been developed, including genetic manipulations to induce gene expression, delivery of biochemical factors, such as triiodothyronine and alpha-adrenergic agonist phenylephrine, induction of cell alignment in 3D tissues, mechanical stress as a mimic of cardiac load and electrical stimulation/pacing or a combination of these...
2017: Frontiers in Cell and Developmental Biology
https://www.readbyqxmd.com/read/28337378/in-vivo-growth-and-responses-to-treatment-of-renal-cell-carcinoma-in-different-environments
#4
Yahya Alhamhoom, Guisheng Zhang, Mingming Gao, Houjian Cai, Dexi Liu
Renal cell carcinoma is the most common type of kidney cancer in adults and is associated with poor prognosis. The hydrodynamic cell delivery technique was employed in this study to establish tumor growth in mouse lung, liver and kidneys. We demonstrate that Renca(Luc) cells exhibit different growth rates and responses to the cancer treatment of 5-florouracil and cytokine gene therapy when growing in different organs. The tumor growth rate was faster in the kidneys compared to that in the lung and liver. The liver is the second-best organ in support of tumor growth...
2017: American Journal of Cancer Research
https://www.readbyqxmd.com/read/28337020/delivery-technologies-for-genome-editing
#5
REVIEW
Hao Yin, Kevin J Kauffman, Daniel G Anderson
With the recent development of CRISPR technology, it is becoming increasingly easy to engineer the genome. Genome-editing systems based on CRISPR, as well as transcription activator-like effector nucleases (TALENs) and zinc-finger nucleases (ZFNs), are becoming valuable tools for biomedical research, drug discovery and development, and even gene therapy. However, for each of these systems to effectively enter cells of interest and perform their function, efficient and safe delivery technologies are needed. This Review discusses the principles of biomacromolecule delivery and gene editing, examines recent advances and challenges in non-viral and viral delivery methods, and highlights the status of related clinical trials...
March 24, 2017: Nature Reviews. Drug Discovery
https://www.readbyqxmd.com/read/28336862/oscillating-magnet-array-based-nanomagnetic-gene-transfection-a-valuable-tool-for-molecular-neurobiology-studies
#6
Mahendran Subramanian, Aimee-Jayne Tyler, Eva Maria Luther, Elena Di Daniel, Jenson Lim, Jon Dobson
To develop treatments for neurodegenerative disorders, it is critical to understand the biology and function of neurons in both normal and diseased states. Molecular studies of neurons involve the delivery of small biomolecules into cultured neurons via transfection to study genetic variants. However, as cultured primary neurons are sensitive to temperature change, stress, and shifts in pH, these factors make biomolecule delivery difficult, particularly non-viral delivery. Herein we used oscillating nanomagnetic gene transfection to successfully transfect SH-SY5Y cells as well as primary hippocampal and cortical neurons on different days in vitro...
January 29, 2017: Nanomaterials
https://www.readbyqxmd.com/read/28336456/small-nanosized-poly-vinyl-benzyl-trimethylammonium-chloride-based-polyplexes-for-sirna-delivery
#7
Bo Lou, Nataliia Beztsinna, Grigoris Mountrichas, Joep B van den Dikkenberg, Stergios Pispas, Wim E Hennink
The success of siRNA gene therapy requires the availability of safe and efficient delivery systems. In the present study, we investigated poly(vinyl benzyl trimethylammonium chloride) (PVTC) and its block copolymer with poly(oligo(ethyleneglycol) methacrylate) (POEGMA) as delivery vector for siRNA. Small polyplexes ranging from 8 to 25nm in diameter were formed in aqueous solution by spontaneous self-assembly of both the homopolymer and block copolymer with siRNA and the formed particles were stable at physiological ionic strength...
March 20, 2017: International Journal of Pharmaceutics
https://www.readbyqxmd.com/read/28336343/viral-delivered-gene-therapy-to-treat-catecholamine-dependent-polymorphic-ventricular-tachycardia-cpvt2-in-mouse-models
#8
Efrat Kurtzwald-Josefson, Dor Yadin, Shiraz Harun-Khun, Maayan Waldman, Dan Aravot, Asher Shainberg, Michael Eldar, Edith Hochhauser, Michael Arad
BACKGROUND: The recessive form of catecholaminergic polymorphic ventricular tachycardia (CPVT2) is caused by mutations in cardiac calsequestrin (CASQ2), leading to protein defficiency. OBJECTIVE: To develop a viral-delivered gene therapy for CPVT2 and determine the relationship between CASQ2 expression and the antiarrhythmic efficacy in a murine model. METHODS: We used a murine model of CPVT2 caused by the D307H human mutation (CASQ2(D307H)) or CASQ2 knock-out (CASQ2(Δ/Δ))...
March 20, 2017: Heart Rhythm: the Official Journal of the Heart Rhythm Society
https://www.readbyqxmd.com/read/28335269/galactosylated-liposomes-for-targeted-co-delivery-of-doxorubicin-vimentin-sirna-to-hepatocellular-carcinoma
#9
Hea Ry Oh, Hyun-Young Jo, James S Park, Dong-Eun Kim, Je-Yoel Cho, Pyung-Hwan Kim, Keun-Sik Kim
The combination of therapeutic nucleic acids and chemotherapeutic drugs has shown great promise for cancer therapy. In this study, asialoglycoprotein receptors (ASGPR) targeting-ligand-based liposomes were tested to determine whether they can co-deliver vimentin siRNA and doxorubicin to hepatocellular carcinoma (HCC) selectively. To achieve this goal, we developed an ASGPR receptor targeted co-delivery system called gal-doxorubicin/vimentin siRNA liposome (Gal-DOX/siRNA-L). The Gal-DOX/siRNA-L was created via electrostatic interaction of galactose linked-cationic liposomal doxorubicin (Gal-DOX-L) on vimentin siRNA...
July 30, 2016: Nanomaterials
https://www.readbyqxmd.com/read/28335259/lipid-nanovectors-to-deliver-rna-oligonucleotides-in-cancer
#10
REVIEW
Virginia Campani, Giuseppina Salzano, Sara Lusa, Giuseppe De Rosa
The growing knowledge on the mechanisms of gene silencing and gene regulation by non-coding RNAs (ncRNA), mainly small interfering RNA (siRNA) and microRNA (miRNA), is providing a significant boost to the development of new therapeutic strategies for the treatment of cancer. However, the design of RNA-based therapeutics is hampered by biopharmaceutical issues, thus requiring the use of suitable delivery strategies. In this regards, lipid nanovectors have been successfully investigated to deliver RNA in different forms of cancer...
July 9, 2016: Nanomaterials
https://www.readbyqxmd.com/read/28335197/cationic-nanoparticles-assembled-from-natural-based-steroid-lipid-for-improved-intracellular-transport-of-sirna-and-pdna
#11
Ruilong Sheng, Xiaoqing Zhuang, Zhao Wang, Amin Cao, Kaili Lin, Julian X X Zhu
Developing new functional biomaterials from biocompatible natural-based resources for gene/drug delivery has attracted increasing attention in recent years. In this work, we prepared a series of cationic nanoparticles (Diosarg-DOPE NPs) by assembly of a natural steroid diosgenin-based cationic lipid (Diosarg) with commercially-available helper lipid 1,2-dioleoyl-sn-glycero-3-phosphorethanolamine (DOPE). These cationic Diosarg-DOPE NPs were able to efficiently bind siRNA and plasmid DNA (pDNA) via electrostatic interactions to form stable, nano-sized cationic lipid nanoparticles instead of lamellar vesicles in aqueous solution...
April 13, 2016: Nanomaterials
https://www.readbyqxmd.com/read/28334782/golgi-retained-cx32-mutants-interfere-with-gene-addition-therapy-for-cmt1x
#12
Styliana Kyriakoudi, Irene Sargiannidou, Alexia Kagiava, Margarita Olympiou, Kleopas A Kleopa
Numerous GJB1 gene mutations cause the X-linked form of Charcot-Marie-Tooth disease (CMT1X). GJB1 encodes connexin32 (Cx32), which forms trans-myelin gap junctions in Schwann cells. Most GJB1 mutations result in loss-of-function mechanisms, supporting the concept of gene replacement therapy. However, interactions between delivered wild type and endogenously expressed mutant Cx32 may potentially occur in the setting of gene replacement therapy. In order to screen for possible interactions of several representative CMT1X mutants with wild type Cx32 that may interfere with functional gap junction formation, we established an in vitro screening method co-expressing in HeLa cells wild type Cx32 and one of eight different Cx32 mutants including A39P, A39V, T55I, R75W, M93V, L143P, N175D and R183S...
February 21, 2017: Human Molecular Genetics
https://www.readbyqxmd.com/read/28334779/targeted-gene-knock-in-by-homology-directed-genome-editing-using-cas9-ribonucleoprotein-and-aav-donor-delivery
#13
Thomas Gaj, Brett T Staahl, Gonçalo M C Rodrigues, Prajit Limsirichai, Freja K Ekman, Jennifer A Doudna, David V Schaffer
Realizing the full potential of genome editing requires the development of efficient and broadly applicable methods for delivering programmable nucleases and donor templates for homology-directed repair (HDR). The RNA-guided Cas9 endonuclease can be introduced into cells as a purified protein in complex with a single guide RNA (sgRNA). Such ribonucleoproteins (RNPs) can facilitate the high-fidelity introduction of single-base substitutions via HDR following co-delivery with a single-stranded DNA oligonucleotide...
March 2, 2017: Nucleic Acids Research
https://www.readbyqxmd.com/read/28334749/ctg-repeat-targeting-oligonucleotides-for-down-regulating-huntingtin-expression
#14
Eman M Zaghloul, Olof Gissberg, Pedro M D Moreno, Lee Siggens, Mattias Hällbrink, Anna S Jørgensen, Karl Ekwall, Rula Zain, Jesper Wengel, Karin E Lundin, C I Edvard Smith
Huntington's disease (HD) is a fatal, neurodegenerative disorder in which patients suffer from mobility, psychological and cognitive impairments. Existing therapeutics are only symptomatic and do not significantly alter the disease progression or increase life expectancy. HD is caused by expansion of the CAG trinucleotide repeat region in exon 1 of the Huntingtin gene (HTT), leading to the formation of mutant HTT transcripts (muHTT). The toxic gain-of-function of muHTT protein is a major cause of the disease...
February 17, 2017: Nucleic Acids Research
https://www.readbyqxmd.com/read/28334507/comparative-study-of-cytotoxicity-and-genotoxicity-of-commercial-jeffamines%C3%A2-and-polyethylenimine-in-cho-k1-cells
#15
Leniher Castan, Cristiano José da Silva, Eduardo Ferreira Molina, Raquel Alves Dos Santos
Jeffamines(®) are a family of polymers containing primary amine groups attached to the extremities of polyether backbone which can be used as biomaterials. They have been used in combination with polyethylenimine (PEI) to improve biocompatibility in drug and gene delivery systems. Despite these facts, very few studies have been done on cytotoxicity and genotoxicity of pure Jeffamines(®) or compared with PEI. The present study aimed to evaluate and compare the cytotoxic and genotoxic effects of Jeffamines(®) and PEI in CHO-K1 cells...
March 23, 2017: Journal of Biomedical Materials Research. Part B, Applied Biomaterials
https://www.readbyqxmd.com/read/28333914/genome-scale-crispr-cas9-knockout-and-transcriptional-activation-screening
#16
Julia Joung, Silvana Konermann, Jonathan S Gootenberg, Omar O Abudayyeh, Randall J Platt, Mark D Brigham, Neville E Sanjana, Feng Zhang
Forward genetic screens are powerful tools for the unbiased discovery and functional characterization of specific genetic elements associated with a phenotype of interest. Recently, the RNA-guided endonuclease Cas9 from the microbial CRISPR (clustered regularly interspaced short palindromic repeats) immune system has been adapted for genome-scale screening by combining Cas9 with pooled guide RNA libraries. Here we describe a protocol for genome-scale knockout and transcriptional activation screening using the CRISPR-Cas9 system...
April 2017: Nature Protocols
https://www.readbyqxmd.com/read/28333149/upregulation-of-mir-665-promotes-apoptosis-and-colitis-in-inflammatory-bowel-disease-by-repressing-the-endoplasmic-reticulum-stress-components-xbp1-and-ormdl3
#17
Manying Li, Shenghong Zhang, Yun Qiu, Yao He, Baili Chen, Ren Mao, Yi Cui, Zhirong Zeng, Minhu Chen
MicroRNAs are critical post-transcriptional regulators of gene expression and key mediators of pathophysiology of inflammatory bowel disease (IBD). This study is aimed to study the role of miR-665 in the progression of IBD. Real-time PCR analysis was used to determine miR-665 expression in 89 freshly isolated IBD samples and dextran sulfate sodium (DSS)-induced colonic mucosal tissues. The role of miR-665 in inducing apoptosis and colitis were examined by Annexin V, TUNEL (terminal deoxynucleotidyl transferase dUTP nick-end labeling) staining, colony formation in vitro and DSS-induced colitis mice model in vivo...
March 23, 2017: Cell Death & Disease
https://www.readbyqxmd.com/read/28333136/targeting-3-phosphoinositide-dependent-protein-kinase-1-associated-with-drug-resistant-renal-cell-carcinoma-using-new-oridonin-analogs
#18
Jiancheng Zhou, Eun-Jin Yun, Wei Chen, Ye Ding, Kaijie Wu, Bin Wang, Chunyong Ding, Elizabeth Hernandez, John Santoyo, Rey-Chen Pong, Haiying Chen, Dalin He, Jia Zhou, Jer-Tsong Hsieh
The current agents used for renal cell carcinoma (RCC) only exhibit the moderate response rate among patients. Development of drug resistance eventually fuels the need of either more potent drugs or new drugs to target the resistant pathways. Oridonin is a diterpenoid isolated from the Chinese medicinal herb Rabdosia rubescens and has been shown to have antitumor activities in many cancers. We previously developed new synthetic methodologies to modify structurally diversified diterpenoids and designed a series of nitrogen-enriched oridonin analogs...
March 23, 2017: Cell Death & Disease
https://www.readbyqxmd.com/read/28332452/editorial-new-trends-in-gene-therapy-multidisciplinary-approaches-to-sirnas-controlled-delivery
#19
EDITORIAL
Anna Angela Barba, Mario Grassi, Gabriele Grassi, Gaetano Lamberti
No abstract text is available yet for this article.
2017: Current Drug Delivery
https://www.readbyqxmd.com/read/28330769/difference-in-the-core-shell-dynamics-of-polyethyleneimine-and-poly-l-lysine-dna-polyplexes
#20
Elina Vuorimaa-Laukkanen, Ekaterina S Lisitsyna, Tiia-Maaria Ketola, Emmanuelle Morin-Pickardat, Huamin Liang, Martina Hanzlíková, Marjo Yliperttula
Electrostatic polymer-DNA complexes (polyplexes) have been widely investigated for DNA delivery, and remarkable differences in transfection efficacy have been seen among the materials. For example, polyethyleneimine (PEI) mediates DNA transfection more effectively than poly(l-lysine) (PLL). Biophysical properties of the polyplexes may explain their different properties in gene delivery. We investigated the structural dynamics in DNA polyplexes, especially the material exchange between the core and shell regions of the PEI and PLL polyplexes...
March 18, 2017: European Journal of Pharmaceutical Sciences
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