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*  Natasha J. Caplen, Ph.D. | Center for Cancer Research - National Cancer Institute
Caplen co-discovered RNA interference (RNAi) in mammalian cells and has pioneered approaches for exploiting this gene ... during which she was involved in some of the first pre-clinical and clinical studies of cationic lipid mediated gene therapy ... where she initially conducted studies investigating hybrid viral vector systems for the delivery of genes. It was while at ... Caplen developed a research interest in the newly identified gene silencing mechanism, RNA interference (RNAi) leading to her ...
  https://ccr.cancer.gov/Genetics-Branch/natasha-j-caplen
*  JoVE | Peer Reviewed Scientific Video Journal - Methods and Protocols
... mediated gene silencing is crucial for the successful application of RNA interference technology to therapeutic interventions. ... To reduce the probability of viral escape mutants, in this study, we constructed lentiviral vector containing vpr and tat shRNA ... Gene therapy offers the promise of curing the HIV-infected patients. Specific, potent, and sustained short hairpin RNA (shRNA)- ... To Construction of P and NP genes eukaryotic expression vectors of Newcastle Disease Virus LaSota strain,study its reverse ...
  https://www.jove.com/visualize?author=Ping-Hua%20Teng%20
*  NOS Antagonism Using Viral Vectors as an Experimental Strategy: Implications for In Vivo Studies of Cardiovascular Control and...
Thomas, C. E., Ehrhardt, A., Kay, M. A. (2003) Progress and problems with the use of viral vectors for gene therapy. Nat Rev ... Nitric oxide (NO) nitric oxide synthase (NOS) viral vectors dominant negative mutants RNA interference cardiovascular control ... However, the cellular mechanisms mediating nitric oxide modulation remain unclear. Upregulation by gene over-expression or down ... Papale, A., Cerovic, M., Brambilla, R. (2009) Viral vector approaches to modify gene expression in the brain. J Neurosci ...
  https://link.springer.com/protocol/10.1007%2F978-1-61737-964-2_15
*  Non-viral Gene Therapy (ebook) by Kazunari Taira | 9784431278795
Several years ago, when the discovery of catalytic RNA was recognized in a public manner,many people asked if new ?elds of ... therapy would soon be available. Although some tentative positive answers wer ... download and read Non-viral Gene Therapy ebook online in PDF format for iPhone, iPad, Android, Computer and Mobile readers. ... 5 Kazunori Kataoka Development of Cationic Block Copolymer-Based Gene Delivery System 1-6 Toshinori Sato Chitosan-Mediated Gene ...
  https://www.ebooks.com/259392/non-viral-gene-therapy/taira-kazunari-kataoka-kazunori-niidome-takuro/
*  Poly(Ester Amine)-Mediated, Aerosol-Delivered Akt1 Small Interfering RNA Suppresses Lung Tumorigenesis - Redorbit
Viral and nonviral vectors are being used for gene therapy. Viral vectors are frequently used because of their intrinsic ... Prospects of RNA interference therapy for cancer. Gene Ther 2006;13:464-477. ... Elbashir SM, Harborth J, Lendeckel W, Yalcin A, Weber K, Tuschl T. Duplexes of 21-nucleotide RNAs mediate RNA interference in ... Many nonviral vectors have been developed for delivering genes to various organs (8). Aerosol-mediated gene delivery is ...
  http://www.redorbit.com/news/health/1459846/polyester_aminemediated_aerosoldelivered_akt1_small_interfering_rna_suppresses_lung_tumorigenesis/
*  publi-2010 | norlux
Cellular toxicity following application of adeno-associated viral vector-mediated RNA interference in the nervous system. ... and to identify and characterize novel molecular targets for targeted therapies, including cell and gene therapy, against high ...
  http://norlux.lu/page/publi-2010
*  This Month in Anesthesiology | Anesthesiology | ASA Publications
The method could open up the possibility of short hairpin RNA-mediated viral vector-based in vivo gene therapy for treatment of ... no significant interference with normal function) or severe (significant interference with normal function). Blood pressure, ... They used a lentivirus that is taken up by cells and expresses the small hairpin RNA to block the production of this voltage- ... What they found is that four out of five designed short hairpin RNA sequences for NaV1.8 were effective in knocking down the ...
  http://anesthesiology.pubs.asahq.org/article.aspx?articleid=1923668
*  Patent US7988668 - Microsyringe for pre-packaged delivery of pharmaceuticals - Google Patents
Gene therapy for neurometabolic disorders. US20040259247. Nov 29, 2001. Dec 23, 2004. Thomas Tuschl. Rna interference mediating ... These therapies include, for example, viral vectors for gene therapy, or protein suppression therapies. As some of these ... Hommel et al., "Local gene knockdown in the brain using viral-mediated RNA interference," Nature Medicine, Dec. 2003; 9(12); ... RNA interference mediated inhibition of adenosine A1 receptor (ADORA1) gene expression using short interfering RNA. ...
  http://www.google.com/patents/US7988668?dq=6,275,983
*  RNA interference as an antiviral approach: targeting HIV-1. - Semantic Scholar
... is an evolutionary conserved gene-silencing mechanism in which 21- to 23-mer double-stranded short interfering RNA (siRNA) ... mediates the sequence-specific degradation of mRNA. The recent discovery that exogenously delivered siRNA can trigger RNAi in ... Crippling of HIV at Multiple Stages with Recombinant Adeno-Associated Viral Mediated RNA Interference. *Oksana V. Gruzdyn, ... Inhibition of HIV-1 by a Lentiviral Vector with a Novel Tat-Inducible Expression System and a Specific Tropism to the Target ...
  https://www.semanticscholar.org/paper/RNA-interference-as-an-antiviral-approach%3A-HIV-1.-Berkhout/49a387aebe620cf73ec5162a4bec56b7e179633f
*  Frontiers | Technical Improvement and Application of Hydrodynamic Gene Delivery in Study of Liver Diseases | Pharmacology
... due to its high efficiency and low risk in triggering immune responses and carcinogenesis in comparison to viral vectors. This ... In addition to DNA, it has also been shown to deliver other substance such as RNA, proteins, synthetic small compounds and even ... In addition to DNA, it has also been shown to deliver other substance such as RNA, proteins, synthetic small compounds and even ... due to its high efficiency and low risk in triggering immune responses and carcinogenesis in comparison to viral vectors. This ...
  https://www.frontiersin.org/articles/10.3389/fphar.2017.00591/full
*  MISSION™ shRNA Library: Next Generation RNA Interference | China-Mainland | Sigma-Aldrich
Viral delivery systems have been extensively studied for gene therapy research and have, thus, undergone numerous modifications ... RNA Interference. Nature, 418, 244-251 (2002). *Elbashir, S.M., et al., Duplexes of 21-nucleotide RNAs mediate RNA interference ... RNA, 9, 493-501 (2003). *Zufferey, R., et al., Multiply attenuated lentiviral vector achieves efficient gene delivery in vivo. ... these vector-based RNAi platforms may be integrated with viral delivery systems allowing the researcher to perform gene ...
  https://www.sigmaaldrich.com/china-mainland/zh/technical-documents/articles/life-science-innovations/mission-shrna-library0.html
*  JCI - CRISPR/Cas9-mediated gene editing ameliorates neurotoxicity in mouse model of Huntington's disease
Allele-specific RNA interference for neurological disease. Gene Ther. 2006;13(6):576-581.. View this article via: PubMed ... Schematics showing the viral vectors used. HA, human influenza hemagglutinin; ITR, inverted terminal repeat; KASH, Klarsicht, ... It remains unknown whether depletion of HTT in the adult brain, regardless of its allele, could be a safe therapy. Here, we ... CRISPR/Cas9-mediated gene editing ameliorates neurotoxicity in mouse model of Huntington's disease. Su Yang,1 Renbao Chang,1,2, ...
  https://www.jci.org/articles/view/92087
*  Silencing the Breast Cancer Resistance Protein Expression and Function in Caco-2 Cells Using Lentiviral Vector-Based Short...
Recently, chemically synthesized RNA interference (RNAi) molecules have been used to silence expression of various genes in ... Hence, the development of more effective and stable gene nuclear silencing RNA-mediating systems is of interest (Priebsch et al ... and cancer gene therapy.. A major disadvantage of this approach is that RNAi synthesized in vitro reduces gene expression only ... These viral vector-based shRNA lentiviral transduction particles for human BCRP were used to knock down the expression of BCRP ...
  http://dmd.aspetjournals.org/content/37/4/737?ijkey=65718061c593d11dea03115b5b7a81c29965dd57&keytype2=tf_ipsecsha
*  Dose optimization for long-term rAAV-mediated RNA interference in the nigrostriatal projection neurons - Infoscience
... mediated gene knockdown is a powerful tool for targeted gene silencing and an emerging novel therapeutic strategy. Recent ... reported unexpected toxicity after utilizing viral-mediated shRNA knockdown in vivo. Thus, it is currently unclear whether ... At high titers, not only the shTH vectors but also the scrambled and green fluorescence protein (GFP)-only controls caused cell ... Our results have implications for both mechanistic and therapeutic studies utilizing long-term shRNA-mediated gene silencing in ...
  https://infoscience.epfl.ch/record/142485?ln=en
*  Books - Lane Medical Library - Stanford University School of Medicine
Therapeutic regulation of gene expression in the inner ear using RNA interference / Maeda, Y., Smith, R.J.H. -- Gene therapy of ... Adeno-Associated Viral Vectors for Gene Delivery to the Nervous System -- Lentiviral Vectors for Gene Delivery to the Nervous ... Nanoparticle-mediated gene delivery to the lung / Rajagopal Ramesh. Retroviral-mediated gene therapy for the differentiation of ... Gene therapy for hepatitis C virus infection -- Gene therapy for HIV-1 infection -- Gene therapy for respiratory viral ...
  https://lane.stanford.edu/biomed-resources/eb.html?a=g
*  Short Hairpin RNA-mediated Selective Knockdown of NaV1.8 Tetrodotoxin-resistant Voltage-gated Sodium Channel in Dorsal Root...
... opening up the exciting possibility of a shRNA-mediated viral vector-based in vivo gene therapy for the currently difficult to ... RNA interference is a posttranscriptional gene silencing mechanism where a short double-stranded RNA guides the recognition and ... Aoki Y, Cioca DP, Oidaira H, Kamiya J, Kiyosawa K: RNA interference may be more potent than antisense RNA in human cancer cell ... Several factors that may influence the efficiency of RNA interference have been reported,27,28 e.g. , the choice of target site ...
  http://anesthesiology.pubs.asahq.org/article.aspx?articleid=1923738
*  Inhibition of hepatitis B virus expression and replication by RNA interference
... of gene expression. Recently, a new vector system called pSUPER (suppression of endogenous RNA), which directs the synthesis of ... viral transcripts and proteins even in the absence of active viral replication makes it a good candidate as an adjuvant therapy ... 4 This process is mediated by 21 to 23 nucleotides, called small interfering RNAs (siRNA), cleaved from dsRNA. Although first ... RNA interference (RNAi) is the process of sequence-specific gene silencing, initiated by double-stranded RNA (dsRNA) that is ...
  http://www.natap.org/2003/hepUpdate/day3.htm
*  JoVE | Peer Reviewed Scientific Video Journal - Methods and Protocols
Medicine, Issue 49, Adeno-associated virus, viral vectors, gene transfer, gene expression, liver cancer, gene therapy ... RNA interference, RNAi, double stranded RNA, dsRNA, double gene knockdown, vitellogenin gene, vg, ultraspiracle gene, usp, ... Here we describe a stable transduction protocol using lentivirus-mediated gene delivery. The lentiviral vector system is ... Gene delivery system for the hair follicle using viral vectors or liposomes for gene targeting to the various cell types in the ...
  https://www.jove.com/visualize/abstract/25238230/hbx-inhibits-cyp2e1-gene-expression-via-downregulating-hnf4-human
*  Patent US8105652 - Methods of making decomposable thin films of polyelectrolytes and uses thereof - Google Patentsuche
Bass, "RNA Interference: The Short Answer," Nature 411, 428-29, 2001.. 7. Behr, "Synthetic Gene-Transfer Vectors" Acc. Chem. ... Schaffer, et al., "Vector Unpacking as a Potential Barrier for Receptor-Mediated Polyplex Gene Delivery" Biotechnol. Bioeng.67 ... Anderson, "Human Gene Therapy" Nature, 392: 25-30, 1996.. 2. Anderson, et al., "Biodegradation and Biocompatibility of PLA and ... Brazeau, et al., "In Vitro Myotoxicity of Selected Cationic Macromolecules Used in Non-Viral Gene Delivery" Pharm. Res. 15: 680 ...
  http://www.google.de/patents/US8105652
*  References - Muscular Dystrophy - Fitness VIP
Xiao, W., Chirmule, N., Berta, S.C., McCullough, B., Gao, G., Wilson, J.M. (1999). Gene therapy vectors based on adeno- ... Modulation of glycogen synthesis by RNA interference: Towards a new therapeutic approach for glycogenosis type II. Hum Mol ... Correction of multiple striated muscles in murine Pompe disease through adeno-associated virus-mediated gene therapy. Mol Ther ... of factor IX gene transfer to skeletal muscle in murine and canine hemophilia B models by adeno-associated viral vector ...
  https://www.fitness-vip.com/muscular-dystrophy/references-omv.html
*  JoVE | Peer Reviewed Scientific Video Journal - Methods and Protocols
DNA Vector-based RNA Interference to Study Gene Function in Cancer. Authors: Daniel B. Stovall, Meimei Wan, Qiang Zhang, ... RNAi has become a powerful research tool in gene function studies. Compared to genetic deletion, RNAi-mediated gene silencing ... Its use in combination with genetically engineered viral vectors, such as lentivirus, facilitates high efficiencies of shRNA ... New therapies for two common prostate diseases, prostate cancer (PrCa) and benign prostatic hyperplasia (BPH), depend ...
  https://www.jove.com/visualize/abstract/20657647/mmp-9-upar-cathepsin-b-silencing-downregulate-integrins-human-glioma
*  JoVE | Peer Reviewed Scientific Video Journal - Methods and Protocols
In addition to chemical treatments, genetic methodologies such as RNA-interference or gene delivery via lentiviral vectors can ... which have been indispensable for defining viral gene requirements and the role of immune responses. However, it is impossible ... Thus, these results indicate that JCTH-4 in combination with TAM could be used as a safe and very potent anti-cancer therapy ... We present the synthesis of a highly stable protein hydrogel mediated by a split-intein-catalyzed protein trans-splicing ...
  https://www.jove.com/visualize/abstract/23667462/a-highly-productive-whole-cell-dera-chemoenzymatic-process-for
*  2014 Abstracts
207 RNA interference-mediated reversal of metallothionein-dependent cisplatin resistance using reducible poly(oligo-D-arginine ... 501 Non-Viral Delivery of Gene HPV-16 E7 into Mammalian Cells. Presenter: F Dorkoosh, Tehran Univ, Iran. Authors: M Dinarvand, ... 178 Development of Modified Pectic Galactan Nano-System for Targeted Gene Therapy. Presenter: N Buaron, Ben-Gurion University ... as more efficient vectors for in vivo transfection of photoreceptors and RPE cells than adeno-associated viral vectors. ...
  https://www.controlledreleasesociety.org/MEETINGS/ARCHIVES/2014ANNUALMEETING/ABSTRACTS/Pages/2014Abstracts.aspx
*  JoVE | Peer Reviewed Scientific Video Journal - Methods and Protocols
Immunology, Issue 82, Canine Adenovirus, viral vector, vaccination, central nervous system, gene therapy ... RNA interference, RNAi, double stranded RNA, dsRNA, double gene knockdown, vitellogenin gene, vg, ultraspiracle gene, usp, ... Here we describe a stable transduction protocol using lentivirus-mediated gene delivery. The lentiviral vector system is ... Adenovirus (Ad) derived vectors have been widely used for short or long-term gene transfer, both for gene therapy and vaccine ...
  https://www.jove.com/visualize/abstract/25275604/the-actin-binding-protein-adseverin-regulates-osteoclastogenesis
*  Protocols and Video Articles Authored by Brian J. Geiss
Recent studies have suggested that RNA interference (RNAi) has therapeutic potential as a sequence specific inhibitor of viral ... can persistently infect and cause limited damage to mosquito vectors. RNA interference (RNAi) is a mosquito antiviral response ... Ring Around the Ro-sie: RNA-mediated Alterations of PNPase Activity Cell. Mar, 2013 , Pubmed ID: 23540686 Chen et al. ... These results suggest that sofosbuvir may be a candidate for further evaluation as a therapy against ZIKV infection in humans. ...
  https://www.jove.com/author/Brian++J._Geiss