Genomes and Genes
Experts and Doctors on gene transfer techniques in United States
Locale: United States
Topic: gene transfer techniques
Publications286 found, 100 shown here
- BEIER K, Mundell N, Pan Y, Cepko C. Anterograde or Retrograde Transsynaptic Circuit Tracing in Vertebrates with Vesicular Stomatitis Virus Vectors. Curr Protoc Neurosci. 2016;74:1.26.1-27 pubmed publisher..Here, we detail the use of these vectors and provide protocols for propagating virus, changing the surface glycoprotein, and infecting multiple organisms using several injection strategies. ..
- Holst J, Szymczak Workman A, Vignali K, Burton A, Workman C, Vignali D. Generation of T-cell receptor retrogenic mice. Nat Protoc. 2006;1:406-17 pubmed..We have successfully used this approach to express over 50 different TCRs on several different mouse backgrounds in as little as 6 weeks. ..
- Clement N, Knop D, Byrne B. Large-scale adeno-associated viral vector production using a herpesvirus-based system enables manufacturing for clinical studies. Hum Gene Ther. 2009;20:796-806 pubmed publisher..At least one human clinical trial employing rAAV generated via rHSV helper-assisted replication is poised to commence, highlighting the advances and relevance of this production method. ..
- Cheng J, Zeidan R, Mishra S, Liu A, Pun S, Kulkarni R, et al. Structure-function correlation of chloroquine and analogues as transgene expression enhancers in nonviral gene delivery. J Med Chem. 2006;49:6522-31 pubmed
- Li B, Xie C, Qiu H. Production of selectable marker-free transgenic tobacco plants using a non-selection approach: chimerism or escape, transgene inheritance, and efficiency. Plant Cell Rep. 2009;28:373-86 pubmed publisher..Transgenes in these transgenic plants were able to transmit the transgene into progeny in a similar fashion as those recovered under selection. ..
- Glasgow J, Mikheeva G, Krasnykh V, Curiel D. A strategy for adenovirus vector targeting with a secreted single chain antibody. PLoS ONE. 2009;4:e8355 pubmed publisher..This approach may facilitate Ad targeting using a wide variety of targeting ligands directed towards a variety of cellular receptors. ..
- Bilovocky N, Romito Digiacomo R, Murcia C, Maricich S, Herrup K. Factors in the genetic background suppress the engrailed-1 cerebellar phenotype. J Neurosci. 2003;23:5105-12 pubmed..In support of this, C57BL/6-En1-/- embryos that are also En2+/- lack a cerebellum and caudal midbrain: a phenotype identical to 129/Sv-En1-/- mice. ..
- El Amouri S, Zhu H, Yu J, Marr R, Verma I, Kindy M. Neprilysin: an enzyme candidate to slow the progression of Alzheimer's disease. Am J Pathol. 2008;172:1342-54 pubmed publisher..These data suggest that restoring NEP levels in the brain at the early stages of AD is an effective strategy to prevent or attenuate disease progression. ..
- Srinivasakumar N, Schuening F. A lentivirus packaging system based on alternative RNA transport mechanisms to express helper and gene transfer vector RNAs and its use to study the requirement of accessory proteins for particle formation and gene delivery. J Virol. 1999;73:9589-98 pubmed..The combination/mixed packaging system was comparable to the other systems for production of virus stocks, suggesting that this design may prove to be safer for the eventual deployment of lentivirus vectors for therapeutic purposes. ..
- Zhong Z, Tsukada S, Rehman H, Parsons C, Theruvath T, Rippe R, et al. Inhibition of transforming growth factor-beta/Smad signaling improves regeneration of small-for-size rat liver grafts. Liver Transpl. 2010;16:181-90 pubmed publisher..Together, these data show that TGF-beta is responsible, at least in part, for the defective liver regeneration in small-for-size grafts by activating the Smad signaling pathway. ..
- Delfin D, Xu Y, Schill K, Mays T, Canan B, Zang K, et al. Sustaining cardiac claudin-5 levels prevents functional hallmarks of cardiomyopathy in a muscular dystrophy mouse model. Mol Ther. 2012;20:1378-83 pubmed publisher..This experiment demonstrates that claudin-5 may represent a novel treatment target for prevention of heart failure. ..
- Blanco Centurion C, Liu M, Konadhode R, Pelluru D, Shiromani P. Effects of orexin gene transfer in the dorsolateral pons in orexin knockout mice. Sleep. 2013;36:31-40 pubmed publisher..In contrast, GFP gene transfer worsened narcoleptic symptoms compared to the no-rAAV orexin KO group. Orexin gene transfer into the dorsolateral pontine neurons can control cataplexy attacks and modestly improve wake maintenance. ..
- Murray S, Santangelo K, Bertone A. Evaluation of early cellular influences of bone morphogenetic proteins 12 and 2 on equine superficial digital flexor tenocytes and bone marrow-derived mesenchymal stem cells in vitro. Am J Vet Res. 2010;71:103-14 pubmed publisher..Targeted equine SDFTNs may respond to BMP12 with improved tenocyte morphology and without mineralization, as seen with BMP2. Bone marrow-derived mesenchymal stem cells may be able to serve as a cell delivery method for BMP12. ..
- Camp E, Wang C, Little E, Watson P, Pirollo K, Rait A, et al. Transferrin receptor targeting nanomedicine delivering wild-type p53 gene sensitizes pancreatic cancer to gemcitabine therapy. Cancer Gene Ther. 2013;20:222-8 pubmed publisher..This approach has the potential to be translated into a new, more effective therapy for pancreatic cancer. Further optimization is ongoing, moving towards a Phase 1B/2 clinical trial. ..
- Baker R, Haendel M, Swanson B, Shambaugh J, Micales B, Lyons G. In vitro preselection of gene-trapped embryonic stem cell clones for characterizing novel developmentally regulated genes in the mouse. Dev Biol. 1997;185:201-14 pubmed..These results also highlight the utility of ES cell differentiation in vitro as a method with which to study the molecular mechanisms regulating the specification and commitment of a variety of cell and tissue types. ..
- Yamazato M, Yamazato Y, Sun C, Diez Freire C, Raizada M. Overexpression of angiotensin-converting enzyme 2 in the rostral ventrolateral medulla causes long-term decrease in blood pressure in the spontaneously hypertensive rats. Hypertension. 2007;49:926-31 pubmed..These observations demonstrate that overexpression of ACE2 overcomes its intrinsic decrease in the RVLM and decreases high blood pressure in the spontaneously hypertensive rat. ..
- Jani P, Singh N, Jenkins C, Raghava S, Mo Y, Amin S, et al. Nanoparticles sustain expression of Flt intraceptors in the cornea and inhibit injury-induced corneal angiogenesis. Invest Ophthalmol Vis Sci. 2007;48:2030-6 pubmed..035) at 5 weeks after administration. Albumin nanoparticles are not toxic to the cornea and can express intraceptors for extended periods that are effective in suppressing injury-induced corneal neovascularization. ..
- Wang Y, Pfeifer B. 6-deoxyerythronolide B production through chromosomal localization of the deoxyerythronolide B synthase genes in E. coli. Metab Eng. 2008;10:33-8 pubmed..As determined by MS analysis, average production levels for YW9 were 0.47 (22 degrees C), 0.52 (30 degrees C), and 0.11 (37 degrees C)mg/L. ..
- Matsuda D, Dreher T. In vivo translation studies of plant viral RNAs using reporter genes. Curr Protoc Microbiol. 2005;Chapter 16:Unit 16K.2 pubmed publisher..Inoculum RNA quality and half-life after delivery to the protoplasts should be monitored closely for careful investigation of translational regulation elements. ..
- Karberg M, Guo H, Zhong J, Coon R, Perutka J, Lambowitz A. Group II introns as controllable gene targeting vectors for genetic manipulation of bacteria. Nat Biotechnol. 2001;19:1162-7 pubmed publisher..Because of their wide host range, mobile group II introns should be useful for genetic engineering and functional genomics in a wide variety of bacteria...
- Andino L, Ryder D, Shapiro A, Matheny M, Zhang Y, Judge M, et al. POMC overexpression in the ventral tegmental area ameliorates dietary obesity. J Endocrinol. 2011;210:199-207 pubmed publisher..In conclusion, these data indicate a role for POMC overexpression within the VTA reward center to combat HF-induced obesity. ..
- Shin J, Pan X, Hakim C, Yang H, Yue Y, Zhang K, et al. Microdystrophin ameliorates muscular dystrophy in the canine model of duchenne muscular dystrophy. Mol Ther. 2013;21:750-7 pubmed publisher..Our results have established the proof-of-concept for microdystrophin therapy in dystrophic muscles of large mammals and set the stage for clinical trial in human patients. ..
- Sorrentino N, Maffia V, Strollo S, Cacace V, Romagnoli N, Manfredi A, et al. A Comprehensive Map of CNS Transduction by Eight Recombinant Adeno-associated Virus Serotypes Upon Cerebrospinal Fluid Administration in Pigs. Mol Ther. 2016;24:276-286 pubmed publisher..Therefore, our data are instrumental for the clinical evaluation of these rAAV vectors in human neurodegenerative diseases. ..
- Ziady A, Kelley T, Milliken E, Ferkol T, Davis P. Functional evidence of CFTR gene transfer in nasal epithelium of cystic fibrosis mice in vivo following luminal application of DNA complexes targeted to the serpin-enzyme complex receptor. Mol Ther. 2002;5:413-9 pubmed..Molecular conjugates targeting the serpin-enzyme complex receptor, used to compact plasmid DNA, hold promise for gene therapy of cystic fibrosis. ..
- Venkatachalan S, Bushman J, Mercado J, Sancar F, CHRISTOPHERSON K, Boileau A. Optimized expression vector for ion channel studies in Xenopus oocytes and mammalian cells using alfalfa mosaic virus. Pflugers Arch. 2007;454:155-63 pubmed..Furthermore, we found that expression using pUNIV was significantly enhanced in oocytes and was remarkably long-lasting in both systems...
- Muniappan L, Ozcan S. Induction of insulin secretion in engineered liver cells by nitric oxide. BMC Physiol. 2007;7:11 pubmed..Liver cells can be engineered to produce insulin and insulin secretion can be induced by treatment with L-arginine via the production of nitric oxide. ..
- Kota J, Handy C, Haidet A, Montgomery C, Eagle A, Rodino Klapac L, et al. Follistatin gene delivery enhances muscle growth and strength in nonhuman primates. Sci Transl Med. 2009;1:6ra15 pubmed publisher..Our results, together with the findings in mice, suggest that therapy with AAV1-FS344 may improve muscle mass and function in patients with certain degenerative muscle disorders. ..
- Park Y, Kang E, Kwon O, Hwang T, Park H, Lee J, et al. Ionically crosslinked Ad/chitosan nanocomplexes processed by electrospinning for targeted cancer gene therapy. J Control Release. 2010;148:75-82 pubmed publisher..These results clearly demonstrate that cancer cell-targeted viral transduction by Ad/chitosan-PEG-FA nanocomplexes can be used effectively for metastatic tumor treatment with reduced immune reaction against Ad. ..
- Wang Z, Lisowski L, Finegold M, Nakai H, Kay M, Grompe M. AAV vectors containing rDNA homology display increased chromosomal integration and transgene persistence. Mol Ther. 2012;20:1902-11 pubmed publisher..We conclude that rDNA containing AAV vectors may be superior to conventional vector design for the treatment of genetic diseases, especially those associated with increased hepatocyte replication. ..
- Basheer W, Xiao S, Epifantseva I, Fu Y, Kléber A, Hong T, et al. GJA1-20k Arranges Actin to Guide Cx43 Delivery to Cardiac Intercalated Discs. Circ Res. 2017;121:1069-1080 pubmed publisher..Exogenous GJA1-20k helps to maintain cell-cell coupling in instances of anticipated myocardial ischemia. ..
- Gomez S, Handler A. A Drosophila melanogaster hobo-white(+) vector mediates low frequency gene transfer in D. virilis with full interspecific white(+) complementation. Insect Mol Biol. 1997;6:165-71 pubmed..virilis. The data reaffirms a relatively low transformation vector activity for the hobo transposon in D. virilis; however, nearly full interspecific expression white(+) marker supports its possible function in other species as well...
- Klein R, Hamby M, Gong Y, Hirko A, Wang S, Hughes J, et al. Dose and promoter effects of adeno-associated viral vector for green fluorescent protein expression in the rat brain. Exp Neurol. 2002;176:66-74 pubmed..The increased efficiency obtained with vector elements such as the CBA promoter and the WPRE may enhance the ability to genetically modify larger portions of the brain while requiring smaller doses and volumes. ..
- O Rourke J, Hiraragi H, Urban K, Patel M, Olsen J, Bunnell B. Analysis of gene transfer and expression in skeletal muscle using enhanced EIAV lentivirus vectors. Mol Ther. 2003;7:632-9 pubmed
- Tyurin M, Desai S, Lynd L. Electrotransformation of Clostridium thermocellum. Appl Environ Microbiol. 2004;70:883-90 pubmed..thermocellum that is suitable for molecular manipulations involving either the introduction of genes associated with foreign gene products or knockout of native genes. ..
- Sun Z. Overview of recent advances in molecular cardiology. Can J Cardiol. 2006;22:235-40 pubmed
- McCrink K, Maning J, Vu A, Jafferjee M, Marrero C, Brill A, et al. ?-Arrestin2 Improves Post-Myocardial Infarction Heart Failure via Sarco(endo)plasmic Reticulum Ca2+-ATPase-Dependent Positive Inotropy in Cardiomyocytes. Hypertension. 2017;70:972-981 pubmed publisher..Thus, cardiac ?arr2 gene transfer might be a novel, safe positive inotropic therapy for both acute and chronic post-MI heart failure. ..
- Guy J, Qi X, Pallotti F, Schon E, Manfredi G, Carelli V, et al. Rescue of a mitochondrial deficiency causing Leber Hereditary Optic Neuropathy. Ann Neurol. 2002;52:534-42 pubmed..Restoration of respiration by allotopic expression opens the door for gene therapy of Leber Hereditary Optic Neuropathy. ..
- Qi X, Lewin A, Sun L, Hauswirth W, Guy J. SOD2 gene transfer protects against optic neuropathy induced by deficiency of complex I. Ann Neurol. 2004;56:182-91 pubmed..Rescue of this animal model supports a critical role for oxidative injury in disorders with complex I deficiency and shows that a respiratory deficit may be effectively treated in mammals, thus offering hope to patients. ..
- TAYLOR J, Vojtech L, Bahner I, Kohn D, Laer D, Russell D, et al. Foamy virus vectors expressing anti-HIV transgenes efficiently block HIV-1 replication. Mol Ther. 2008;16:46-51 pubmed..These results suggest that there are potential benefits of using FV vectors in HIV gene therapy. ..
- Heller K, Montgomery C, Janssen P, Clark K, Mendell J, Rodino Klapac L. AAV-mediated overexpression of human ?7 integrin leads to histological and functional improvement in dystrophic mice. Mol Ther. 2013;21:520-5 pubmed publisher..This therapeutic approach demonstrates promise as a viable treatment for DMD with further implications for other forms of muscular dystrophy. ..
- Sahoo D, Stork J, DeBolt S, Maiti I. Manipulating cellulose biosynthesis by expression of mutant Arabidopsis proM24::CESA3(ixr1-2) gene in transgenic tobacco. Plant Biotechnol J. 2013;11:362-72 pubmed publisher..Moreover, the introduction of M24-AtCESA3(ixr1-2) caused aberrant spatial distribution of lignified secondary cell wall tissue and a reduction in the zone occupied by parenchyma cells. ..
- Nair S, Lindeman R, Pelegri F. In vitro oocyte culture-based manipulation of zebrafish maternal genes. Dev Dyn. 2013;242:44-52 pubmed publisher..These manipulations extend the potential to carry out genetic and imaging studies of zebrafish maternal genes during the egg-to-embryo transition. ..
- Deng W, Sakurai K, Kolandaivelu S, Kolesnikov A, Dinculescu A, Li J, et al. Cone phosphodiesterase-6?' restores rod function and confers distinct physiological properties in the rod phosphodiesterase-6?-deficient rd10 mouse. J Neurosci. 2013;33:11745-53 pubmed publisher..Together, these results demonstrate that cone PDE6?' can functionally substitute for rod PDE?? in vivo, conferring treated rods with distinct physiological properties. ..
- Wilson S, Yeomans D. Virally mediated delivery of enkephalin and other neuropeptide transgenes in experimental pain models. Ann N Y Acad Sci. 2002;971:515-21 pubmed..These results raise the possibility that herpes-mediated, gene-based approaches to treat chronic pain states may be useful in therapy of chronic pain in humans. ..
- Kyrkanides S, Miller J, Tallents R, Brouxhon S, Centola G, Olschowka J. Intraperitoneal inoculation of Sandhoff mouse neonates with an HIV-1 based lentiviral vector exacerbates the attendant neuroinflammation and disease phenotype. J Neuroimmunol. 2007;188:39-47 pubmed..We observed an increase and acceleration in the presence of MHC-II and CD45+ cells in their brains, along with neuroinflammation, but not in control heterozygous or wild type littermates that also received the same treatment. ..
- Igarashi T, Finet J, Takeuchi A, Fujino Y, Strom M, Greener I, et al. Connexin gene transfer preserves conduction velocity and prevents atrial fibrillation. Circulation. 2012;125:216-25 pubmed publisher..In the AF group, both Cx40 and Cx43 gene transfer improved conduction and reduced AF relative to controls. Connexin gene therapy preserved atrial conduction and prevented AF. ..
- Zhang Z, Galileo D. Retroviral transfer of antisense integrin alpha6 or alpha8 sequences results in laminar redistribution or clonal cell death in developing brain. J Neurosci. 1998;18:6928-38 pubmed..Also alpha8 integrin-substrate interactions may suppress early programmed cell death in premigratory and migratory neuroblasts. ..
- Chao J, Kato K, Zhang J, Dobrzynski E, Wang C, Agata J, et al. Human adrenomedullin gene delivery protects against cardiovascular remodeling and renal injury. Peptides. 2001;22:1731-7 pubmed..These findings provide new insights into the role of AM in cardiovascular and renal function. ..
- Sebestyen M, Budker V, Budker T, Subbotin V, Zhang G, Monahan S, et al. Mechanism of plasmid delivery by hydrodynamic tail vein injection. I. Hepatocyte uptake of various molecules. J Gene Med. 2006;8:852-73 pubmed
- Rhee M, Kim J, Qian Y, Ingram L, Shanmugam K. Development of plasmid vector and electroporation condition for gene transfer in sporogenic lactic acid bacterium, Bacillus coagulans. Plasmid. 2007;58:13-22 pubmed publisher..coagulans strain P4-102B using the plasmids pNW33N or pMSR10 was about 1.5x10(16) per mole of DNA. The availability of shuttle vectors and an electroporation method is expected to aid in genetic and metabolic engineering of B. coagulans...
- Harkey M, Asano A, Zoulas M, Torok Storb B, Nagashima J, Travis A. Isolation, genetic manipulation, and transplantation of canine spermatogonial stem cells: progress toward transgenesis through the male germ-line. Reproduction. 2013;146:75-90 pubmed publisher..These findings help to set the stage for generation of transgenic canine models via SSC transplantation. ..
- Pozsgai E, Griffin D, Heller K, Mendell J, Rodino Klapac L. β-Sarcoglycan gene transfer decreases fibrosis and restores force in LGMD2E mice. Gene Ther. 2016;23:57-66 pubmed publisher..This study demonstrates the potential for gene replacement to reverse debilitating fibrosis, typical of muscular dystrophy, thereby providing compelling evidence for movement to clinical gene replacement for LGMD2E. ..
- Staehling Hampton K, Jackson P, Clark M, Brand A, Hoffmann F. Specificity of bone morphogenetic protein-related factors: cell fate and gene expression changes in Drosophila embryos induced by decapentaplegic but not 60A. Cell Growth Differ. 1994;5:585-93 pubmed
- Hynes S, Smith L, Richardson D, Kovesdi I, O BRIEN T, Katusic Z. In vivo expression and function of recombinant GTPCH I in the rabbit carotid artery. Am J Physiol Heart Circ Physiol. 2004;286:H570-4 pubmed..However, under physiological conditions, it appears that this increase does not affect nitric oxide production in endothelial cells of the carotid artery. ..
- Pliakas A, Carlson R, Neve R, Konradi C, Nestler E, Carlezon W. Altered responsiveness to cocaine and increased immobility in the forced swim test associated with elevated cAMP response element-binding protein expression in nucleus accumbens. J Neurosci. 2001;21:7397-403 pubmed..Exposure to the FST itself dramatically increased CREB function in the NAc. These findings raise the possibility that CREB-mediated transcription within the NAc regulates dysphoric states. ..
- Lobo B, Vetro J, Suich D, Zuckermann R, Middaugh C. Structure/function analysis of peptoid/lipitoid:DNA complexes. J Pharm Sci. 2003;92:1905-18 pubmed
- Blenman K, Duan B, Xu Z, Wan S, Atkinson M, Flotte T, et al. IL-10 regulation of lupus in the NZM2410 murine model. Lab Invest. 2006;86:1136-48 pubmed..These results also reinforce the notion that IL-10 exerts multiple functions and commend caution in equating high levels of IL-10 and increased pathogenesis in systemic autoimmunity. ..
- Zachos T, Diggs A, Weisbrode S, Bartlett J, Bertone A. Mesenchymal stem cell-mediated gene delivery of bone morphogenetic protein-2 in an articular fracture model. Mol Ther. 2007;15:1543-50 pubmed..002). We were successful in achieving repair of both bone and cartilage in vivousing direct stem cell injection. Our data suggests that BMP2 augmentation might be critically important in achieving this effect. ..
- Zhang G, Mohammad H, Peper B, Raja S, Wilson S, Sweitzer S. Enhanced peripheral analgesia using virally mediated gene transfer of the mu-opioid receptor in mice. Anesthesiology. 2008;108:305-13 pubmed publisher..This gene therapy approach may provide an innovative strategy to enhance peripheral opioid analgesia for the treatment of pain in humans, thereby minimizing centrally mediated opioid side effects such as sedation and addiction. ..
- Wiethoff C, Smith J, Koe G, Middaugh C. The potential role of proteoglycans in cationic lipid-mediated gene delivery. Studies of the interaction of cationic lipid-DNA complexes with model glycosaminoglycans. J Biol Chem. 2001;276:32806-13 pubmed..Titration calorimetry reveals an exothermic heat for the interaction glycosaminoglycans with CLDCs at higher ionic strength. These results are consistent with the direct involvement of proteoglycans in transfection. ..
- Heinrich G, Pagtakhan C. Both 5' and 3' flanks regulate Zebrafish brain-derived neurotrophic factor gene expression. BMC Neurosci. 2004;5:19 pubmed..HCS1, a highly conserved sequence in 5' exon 1c, restricts expression to primary sensory neurons. The tools are now available for detailed genetic and molecular analyses of zebrafish BDNF gene expression. ..
- Dong B, Silverman R, Kandel E. A natural human retrovirus efficiently complements vectors based on murine leukemia virus. PLoS ONE. 2008;3:e3144 pubmed publisher..The similarity between the MLV and XMRV genomes suggests a possibility that the two viruses may interact when present in the same cell...
- McDevitt R, Hiroi R, MacKenzie S, Robin N, Cohn A, Kim J, et al. Serotonin 1B autoreceptors originating in the caudal dorsal raphe nucleus reduce expression of fear and depression-like behavior. Biol Psychiatry. 2011;69:780-7 pubmed publisher..Because systemic pharmacologic treatment with a 5-HT(1B) agonist facilitates reductions in fear, 5-HT(1B) receptors may be a target for the treatment of certain anxiety disorders. ..
- Rossmiller B, Mao H, Lewin A. Gene therapy in animal models of autosomal dominant retinitis pigmentosa. Mol Vis. 2012;18:2479-96 pubmed..We conclude that combinatorial approaches have the greatest promise for success. ..
- Alvarez R, Sill M, Davidson S, Muller C, Bender D, DeBernardo R, et al. A phase II trial of intraperitoneal EGEN-001, an IL-12 plasmid formulated with PEG-PEI-cholesterol lipopolymer in the treatment of persistent or recurrent epithelial ovarian, fallopian tube or primary peritoneal cancer: a gynecologic oncology group s. Gynecol Oncol. 2014;133:433-8 pubmed publisher..89 and 9.17months, respectively. EGEN-001 at the dose and schedule evaluated was associated with some but limited activity and was seemingly less tolerated in platinum resistant recurrent ovarian cancer patients. ..
- Salem M, Gillanders W, Kadima A, El Naggar S, Rubinstein M, Demcheva M, et al. Review: novel nonviral delivery approaches for interleukin-12 protein and gene systems: curbing toxicity and enhancing adjuvant activity. J Interferon Cytokine Res. 2006;26:593-608 pubmed..This article discusses the potential capabilities of these nonvirus-based IL-12 delivery systems in different disease settings, including allergy, infection, and cancer. ..
- Rodino Klapac L, Janssen P, Montgomery C, Coley B, Chicoine L, Clark K, et al. A translational approach for limb vascular delivery of the micro-dystrophin gene without high volume or high pressure for treatment of Duchenne muscular dystrophy. J Transl Med. 2007;5:45 pubmed
- Cooper M, Nayak S, Hoffman B, Terhorst C, Cao O, Herzog R. Improved induction of immune tolerance to factor IX by hepatic AAV-8 gene transfer. Hum Gene Ther. 2009;20:767-76 pubmed publisher..IX largely independent of genetic factors. A comparison with other studies suggests that additional parameters besides plateau levels of F.IX expression contributed to the improved success rate of tolerance induction. ..
- Howard P, Howard T, Maurer R. Generation of mice with a conditional allele for Ift172. Transgenic Res. 2010;19:121-6 pubmed publisher..We have confirmed the phenotype of the disrupted allele by using CRE expression directed by the prx1 enhancer to disrupt the conditional Ift172 allele in the developing limb...
- Zhang G, Wooddell C, Hegge J, Griffin J, Huss T, Braun S, et al. Functional efficacy of dystrophin expression from plasmids delivered to mdx mice by hydrodynamic limb vein injection. Hum Gene Ther. 2010;21:221-37 pubmed publisher..These studies show promise for the use of HLV injections to deliver therapeutic doses of full-length dystrophin-expressing plasmids for long-lasting protection of skeletal muscles in patients with DMD. ..
- Bhoopathi P, Chetty C, Gogineni V, Gujrati M, Dinh D, Rao J, et al. MMP-2 mediates mesenchymal stem cell tropism towards medulloblastoma tumors. Gene Ther. 2011;18:692-701 pubmed publisher..In summary, we conclude that hUCBSCs can integrate into human medulloblastoma after local delivery and that MMP-2 expression by the tumor cells mediates this response through the SDF1/CXCR4 axis. ..
- Klueh U, Dorsky D, Kreutzer D. Enhancement of implantable glucose sensor function in vivo using gene transfer-induced neovascularization. Biomaterials. 2005;26:1155-63 pubmed
- Staecker H, Praetorius M, Baker K, Brough D. Vestibular hair cell regeneration and restoration of balance function induced by math1 gene transfer. Otol Neurotol. 2007;28:223-31 pubmed..Molecular replacement of math1 may provide a therapeutic means of restoring vestibular function related to vestibular hair cell loss. ..
- Xu L, Betker J, Yin H, Anchordoquy T. Ligands located within a cholesterol domain enhance gene delivery to the target tissue. J Control Release. 2012;160:57-63 pubmed publisher..These results demonstrate that the microenvironment of the ligand can affect gene delivery to tumors, and show that ligand-mediated delivery can be enhanced by locating targeting ligands within a cholesterol domain. ..
- Elmallah M, Falk D, Lane M, Conlon T, Lee K, Shafi N, et al. Retrograde gene delivery to hypoglossal motoneurons using adeno-associated virus serotype 9. Hum Gene Ther Methods. 2012;23:148-56 pubmed publisher..We conclude that AAV9 can retrogradely infect a significant portion of a given motoneuron pool in normal and dystrophic mice, and that its transduction efficiency is approximately 30% of what can be achieved with CT-?. ..
- Sun W, Fletcher D, van Heeckeren R, Davis P. Non-covalent ligand conjugation to biotinylated DNA nanoparticles using TAT peptide genetically fused to monovalent streptavidin. J Drug Target. 2012;20:678-90 pubmed publisher..In vivo studies showed that TAT modified DNA NPs mediated equal level of gene transfer to the mouse airways via the luminal route compared to unmodified DNA NPs. ..
- Juengst E. Can enhancement be distinguished from prevention in genetic medicine?. J Med Philos. 1997;22:125-42 pubmed..2) Legitimate preventive genetic health care should be limited to efforts to defend people from attack by these more robust pathological entities, rather than changing their bodies to evade social injustices. ..
- Lai Y, Yue Y, Liu M, Ghosh A, Engelhardt J, Chamberlain J, et al. Efficient in vivo gene expression by trans-splicing adeno-associated viral vectors. Nat Biotechnol. 2005;23:1435-9 pubmed..This demonstrates the use of trans-splicing vectors to efficiently express a large therapeutic structural protein. This strategy should be applicable to other large therapeutic genes or large transcription regulatory elements. ..
- Zhong L, Zhao W, Wu J, Maina N, Han Z, Srivastava A. Adeno-associated virus-mediated gene transfer in hematopoietic stem/ progenitor cells as a therapeutic tool. Curr Gene Ther. 2006;6:683-98 pubmed
- Yin H, Chao L, Chao J. Kallikrein/kinin protects against myocardial apoptosis after ischemia/reperfusion via Akt-glycogen synthase kinase-3 and Akt-Bad.14-3-3 signaling pathways. J Biol Chem. 2005;280:8022-30 pubmed..14-3-3 signaling pathways. In conclusion, kallikrein/kinin protects against cardiomyocyte apoptosis in vivo and in vitro via Akt-Bad.14-3-3 and Akt-GSK-3beta-caspase-3 signaling pathways. ..
- Clark M, Sexton T, McClain M, Root D, Kohen R, Neumaier J. Overexpression of 5-HT1B receptor in dorsal raphe nucleus using Herpes Simplex Virus gene transfer increases anxiety behavior after inescapable stress. J Neurosci. 2002;22:4550-62 pubmed..Because these tests are sensitive to increases in anxiety-like behavior, our results suggest that overactivity of 5-HT(1B) autoreceptors in DRN neurons may be an important mediator of pathological responses to stressful events. ..
- Sauka Spengler T, Barembaum M. Gain- and loss-of-function approaches in the chick embryo. Methods Cell Biol. 2008;87:237-56 pubmed publisher..We routinely overexpress constructs containing 3-4 kb inserts and coharboring a GFP or RFP reporter whose translation is initiated from an internal ribosomal entry site (IRES), thus allowing easy detection of the electroporated cells. ..
- Nande R, Di Benedetto A, Aimola P, de Carlo F, Carper M, Claudio C, et al. Targeting a newly established spontaneous feline fibrosarcoma cell line by gene transfer. PLoS ONE. 2012;7:e37743 pubmed publisher..More gene transfer studies should be conducted in order to understand if these viral vectors could be applicable regardless the origin (spontaneous vs. vaccine induced) of feline fibrosarcomas...
- MacLaren R, Groppe M, Barnard A, Cottriall C, Tolmachova T, Seymour L, et al. Retinal gene therapy in patients with choroideremia: initial findings from a phase 1/2 clinical trial. Lancet. 2014;383:1129-37 pubmed publisher..UK Department of Health and Wellcome Trust. ..