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MeSH Review

Gene Therapy

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Disease relevance of Gene Therapy


Psychiatry related information on Gene Therapy


High impact information on Gene Therapy

  • Clinical trials of gene therapy for ADA deficiency have been initiated [9].
  • In gene therapy experiments, the rat NIS gene has been transduced into various types of human cells, which then exhibited active iodide transport and became susceptible to destruction with radioiodide [10].
  • Our results also indicate therapeutic strategies for the treatment of HCC including use of SOCS-1 in gene therapy and inhibition of JAK2 by small molecules, such as AG490 [11].
  • These experiments define aspects of adenovirus-mediated gene therapy relevant to CF protocols based on intrapulmonary genetic reconstitution [12].
  • Gene therapy for cholesterol [13].

Chemical compound and disease context of Gene Therapy


Biological context of Gene Therapy


Anatomical context of Gene Therapy


Associations of Gene Therapy with chemical compounds


Gene context of Gene Therapy

  • The functional improvement of dystrophic muscle by myostatin blockade provides a novel, pharmacological strategy for treatment of diseases associated with muscle wasting such as DMD, and circumvents the major problems associated with conventional gene therapy in these disorders [34].
  • Our data suggest that Neurod-Btc gene therapy is a promising regimen to induce islet neogenesis for the treatment of insulin-dependent diabetes [35].
  • IL-4 gene therapy for collagen arthritis suppresses synovial IL-17 and osteoprotegerin ligand and prevents bone erosion [36].
  • Restored insulin-sensitivity in IRS-1-deficient mice treated by adenovirus-mediated gene therapy [37].
  • These findings indicate that it may be possible to develop CF therapies (e.g., mini-cDNA constructs for gene therapy) that are tailored to specific disease-causing mutants of CFTR [38].

Analytical, diagnostic and therapeutic context of Gene Therapy


  1. Efficiency of gene transfer for restoration of normal airway epithelial function in cystic fibrosis. Johnson, L.G., Olsen, J.C., Sarkadi, B., Moore, K.L., Swanstrom, R., Boucher, R.C. Nat. Genet. (1992) [Pubmed]
  2. Gene therapy restores vision in a canine model of childhood blindness. Acland, G.M., Aguirre, G.D., Ray, J., Zhang, Q., Aleman, T.S., Cideciyan, A.V., Pearce-Kelling, S.E., Anand, V., Zeng, Y., Maguire, A.M., Jacobson, S.G., Hauswirth, W.W., Bennett, J. Nat. Genet. (2001) [Pubmed]
  3. Phenotype correction of hemophilia A mice by spliceosome-mediated RNA trans-splicing. Chao, H., Mansfield, S.G., Bartel, R.C., Hiriyanna, S., Mitchell, L.G., Garcia-Blanco, M.A., Walsh, C.E. Nat. Med. (2003) [Pubmed]
  4. Sonic hedgehog myocardial gene therapy: tissue repair through transient reconstitution of embryonic signaling. Kusano, K.F., Pola, R., Murayama, T., Curry, C., Kawamoto, A., Iwakura, A., Shintani, S., Ii, M., Asai, J., Tkebuchava, T., Thorne, T., Takenaka, H., Aikawa, R., Goukassian, D., von Samson, P., Hamada, H., Yoon, Y.S., Silver, M., Eaton, E., Ma, H., Heyd, L., Kearney, M., Munger, W., Porter, J.A., Kishore, R., Losordo, D.W. Nat. Med. (2005) [Pubmed]
  5. Attenuation of seizures and neuronal death by adeno-associated virus vector galanin expression and secretion. Haberman, R.P., Samulski, R.J., McCown, T.J. Nat. Med. (2003) [Pubmed]
  6. Cloning of rat and human inducible penile nitric oxide synthase. Application for gene therapy of erectile dysfunction. Garbán, H., Marquez, D., Magee, T., Moody, J., Rajavashisth, T., Rodríguez, J.A., Hung, A., Vernet, D., Rajfer, J., González-Cadavid, N.F. Biol. Reprod. (1997) [Pubmed]
  7. Gene therapy approaches for the selective killing of cancer cells. Westphal, E.M., Melchner Hv, H. Curr. Pharm. Des. (2002) [Pubmed]
  8. Role of brain-derived neurotrophic factor in eating disorders: recent findings and its pathophysiological implications. Hashimoto, K., Koizumi, H., Nakazato, M., Shimizu, E., Iyo, M. Prog. Neuropsychopharmacol. Biol. Psychiatry (2005) [Pubmed]
  9. Gene therapy of the immune system. Cournoyer, D., Caskey, C.T. Annu. Rev. Immunol. (1993) [Pubmed]
  10. Molecular analysis of the sodium/iodide symporter: impact on thyroid and extrathyroid pathophysiology. De La Vieja, A., Dohan, O., Levy, O., Carrasco, N. Physiol. Rev. (2000) [Pubmed]
  11. SOCS-1, a negative regulator of the JAK/STAT pathway, is silenced by methylation in human hepatocellular carcinoma and shows growth-suppression activity. Yoshikawa, H., Matsubara, K., Qian, G.S., Jackson, P., Groopman, J.D., Manning, J.E., Harris, C.C., Herman, J.G. Nat. Genet. (2001) [Pubmed]
  12. Gene therapy in a xenograft model of cystic fibrosis lung corrects chloride transport more effectively than the sodium defect. Goldman, M.J., Yang, Y., Wilson, J.M. Nat. Genet. (1995) [Pubmed]
  13. Gene therapy for cholesterol. Brown, M.S., Goldstein, J.L., Havel, R.J., Steinberg, D. Nat. Genet. (1994) [Pubmed]
  14. Vesicular stomatitis virus G glycoprotein pseudotyped retroviral vectors: concentration to very high titer and efficient gene transfer into mammalian and nonmammalian cells. Burns, J.C., Friedmann, T., Driever, W., Burrascano, M., Yee, J.K. Proc. Natl. Acad. Sci. U.S.A. (1993) [Pubmed]
  15. Prevention of renovascular and cardiac pathophysiological changes in hypertension by angiotensin II type 1 receptor antisense gene therapy. Martens, J.R., Reaves, P.Y., Lu, D., Katovich, M.J., Berecek, K.H., Bishop, S.P., Raizada, M.K., Gelband, C.H. Proc. Natl. Acad. Sci. U.S.A. (1998) [Pubmed]
  16. Absence of a desmopressin response after therapeutic expression of factor VIII in hemophilia A dogs with liver-directed neonatal gene therapy. Xu, L., Nichols, T.C., Sarkar, R., McCorquodale, S., Bellinger, D.A., Ponder, K.P. Proc. Natl. Acad. Sci. U.S.A. (2005) [Pubmed]
  17. Digitoxin mimics gene therapy with CFTR and suppresses hypersecretion of IL-8 from cystic fibrosis lung epithelial cells. Srivastava, M., Eidelman, O., Zhang, J., Paweletz, C., Caohuy, H., Yang, Q., Jacobson, K.A., Heldman, E., Huang, W., Jozwik, C., Pollard, B.S., Pollard, H.B. Proc. Natl. Acad. Sci. U.S.A. (2004) [Pubmed]
  18. A factor IX-deficient mouse model for hemophilia B gene therapy. Wang, L., Zoppè, M., Hackeng, T.M., Griffin, J.H., Lee, K.F., Verma, I.M. Proc. Natl. Acad. Sci. U.S.A. (1997) [Pubmed]
  19. Long-term control of erythropoietin secretion by doxycycline in mice transplanted with engineered primary myoblasts. Bohl, D., Naffakh, N., Heard, J.M. Nat. Med. (1997) [Pubmed]
  20. Functional amounts of dystrophin produced by skipping the mutated exon in the mdx dystrophic mouse. Lu, Q.L., Mann, C.J., Lou, F., Bou-Gharios, G., Morris, G.E., Xue, S.A., Fletcher, S., Partridge, T.A., Wilton, S.D. Nat. Med. (2003) [Pubmed]
  21. Implantation of genetically engineered fibroblasts into mice: implications for gene therapy. Selden, R.F., Skoskiewicz, M.J., Howie, K.B., Russell, P.S., Goodman, H.M. Science (1987) [Pubmed]
  22. Reversal of impaired wound repair in iNOS-deficient mice by topical adenoviral-mediated iNOS gene transfer. Yamasaki, K., Edington, H.D., McClosky, C., Tzeng, E., Lizonova, A., Kovesdi, I., Steed, D.L., Billiar, T.R. J. Clin. Invest. (1998) [Pubmed]
  23. Direct gene delivery of human tissue kallikrein reduces blood pressure in spontaneously hypertensive rats. Wang, C., Chao, L., Chao, J. J. Clin. Invest. (1995) [Pubmed]
  24. Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1. Ott, M.G., Schmidt, M., Schwarzwaelder, K., Stein, S., Siler, U., Koehl, U., Glimm, H., Kühlcke, K., Schilz, A., Kunkel, H., Naundorf, S., Brinkmann, A., Deichmann, A., Fischer, M., Ball, C., Pilz, I., Dunbar, C., Du, Y., Jenkins, N.A., Copeland, N.G., Lüthi, U., Hassan, M., Thrasher, A.J., Hoelzer, D., von Kalle, C., Seger, R., Grez, M. Nat. Med. (2006) [Pubmed]
  25. Chronic suppression of heart-failure progression by a pseudophosphorylated mutant of phospholamban via in vivo cardiac rAAV gene delivery. Hoshijima, M., Ikeda, Y., Iwanaga, Y., Minamisawa, S., Date, M.O., Gu, Y., Iwatate, M., Li, M., Wang, L., Wilson, J.M., Wang, Y., Ross, J., Chien, K.R. Nat. Med. (2002) [Pubmed]
  26. CII-DC-AdTRAIL cell gene therapy inhibits infiltration of CII-reactive T cells and CII-induced arthritis. Liu, Z., Xu, X., Hsu, H.C., Tousson, A., Yang, P.A., Wu, Q., Liu, C., Yu, S., Zhang, H.G., Mountz, J.D. J. Clin. Invest. (2003) [Pubmed]
  27. Bone marrow extracellular matrix molecules improve gene transfer into human hematopoietic cells via retroviral vectors. Moritz, T., Patel, V.P., Williams, D.A. J. Clin. Invest. (1994) [Pubmed]
  28. Hematopoietic stem cell gene therapy: selecting only the best. Bank, A. J. Clin. Invest. (2003) [Pubmed]
  29. UK scientists test liposome gene therapy technique. Dickson, D. Nature (1993) [Pubmed]
  30. Peroral gene therapy of lactose intolerance using an adeno-associated virus vector. During, M.J., Xu, R., Young, D., Kaplitt, M.G., Sherwin, R.S., Leone, P. Nat. Med. (1998) [Pubmed]
  31. Rhône-Poulenc to focus biotech work on gene therapy. Butler, D. Nature (1994) [Pubmed]
  32. Glucose-stimulated genes and prospects of gene therapy for type I diabetes. Mitanchez, D., Doiron, B., Chen, R., Kahn, A. Endocr. Rev. (1997) [Pubmed]
  33. Transactivation of the metallothionein promoter in cisplatin-resistant cancer cells: a specific gene therapy strategy. Vandier, D., Calvez, V., Massade, L., Gouyette, A., Mickley, L., Fojo, T., Rixe, O. J. Natl. Cancer Inst. (2000) [Pubmed]
  34. Functional improvement of dystrophic muscle by myostatin blockade. Bogdanovich, S., Krag, T.O., Barton, E.R., Morris, L.D., Whittemore, L.A., Ahima, R.S., Khurana, T.S. Nature (2002) [Pubmed]
  35. NeuroD-betacellulin gene therapy induces islet neogenesis in the liver and reverses diabetes in mice. Kojima, H., Fujimiya, M., Matsumura, K., Younan, P., Imaeda, H., Maeda, M., Chan, L. Nat. Med. (2003) [Pubmed]
  36. IL-4 gene therapy for collagen arthritis suppresses synovial IL-17 and osteoprotegerin ligand and prevents bone erosion. Lubberts, E., Joosten, L.A., Chabaud, M., van Den Bersselaar, L., Oppers, B., Coenen-De Roo, C.J., Richards, C.D., Miossec, P., van Den Berg, W.B. J. Clin. Invest. (2000) [Pubmed]
  37. Restored insulin-sensitivity in IRS-1-deficient mice treated by adenovirus-mediated gene therapy. Ueki, K., Yamauchi, T., Tamemoto, H., Tobe, K., Yamamoto-Honda, R., Kaburagi, Y., Akanuma, Y., Yazaki, Y., Aizawa, S., Nagai, R., Kadowaki, T. J. Clin. Invest. (2000) [Pubmed]
  38. Rescuing cystic fibrosis transmembrane conductance regulator (CFTR)-processing mutants by transcomplementation. Cormet-Boyaka, E., Jablonsky, M., Naren, A.P., Jackson, P.L., Muccio, D.D., Kirk, K.L. Proc. Natl. Acad. Sci. U.S.A. (2004) [Pubmed]
  39. Induction of the p16INK4a senescence gene as a new therapeutic strategy for the treatment of rheumatoid arthritis. Taniguchi, K., Kohsaka, H., Inoue, N., Terada, Y., Ito, H., Hirokawa, K., Miyasaka, N. Nat. Med. (1999) [Pubmed]
  40. A20 inhibits cytokine-induced apoptosis and nuclear factor kappaB-dependent gene activation in islets. Grey, S.T., Arvelo, M.B., Hasenkamp, W., Bach, F.H., Ferran, C. J. Exp. Med. (1999) [Pubmed]
  41. Genetic correction of dystrophin deficiency and skeletal muscle remodeling in adult MDX mouse via transplantation of retroviral producer cells. Fassati, A., Wells, D.J., Sgro Serpente, P.A., Walsh, F.S., Brown, S.C., Strong, P.N., Dickson, G. J. Clin. Invest. (1997) [Pubmed]
  42. Correction of renal tubular acidosis in carbonic anhydrase II-deficient mice with gene therapy. Lai, L.W., Chan, D.M., Erickson, R.P., Hsu, S.J., Lien, Y.H. J. Clin. Invest. (1998) [Pubmed]
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