Trans-splicing repair of CD40 ligand deficiency results in naturally regulated correction of a mouse model of hyper-IgM X-linked immunodeficiency (original) (raw)

• Callard, R.E., Armitage, R.J., Fanslow, W.C. & Spriggs, M.K. CD40 ligand and its role in X-linked hyper-IgM syndrome. Immunol. Today 14, 559–564 (1993).
  Article CAS PubMed Google Scholar
• Kroczek, R.A. et al. Defective expression of CD40 ligand on T cells causes “X-linked immunodeficiency with hyper-IgM (HIGM1)”. Immunol. Rev. 138, 39–59 (1994).
  Article CAS PubMed Google Scholar
• Di Santo, J.P., Bonnefoy, J.Y., Gauchat, J.F., Fischer, A. & de Saint, B.G. CD40 ligand mutations in X-linked immunodeficiency with hyper-IgM. Nature 361, 541–543 (1993).
  Article CAS Google Scholar
• Korthauer, U. et al. Defective expression of T-cell CD40 ligand causes X-linked immunodeficiency with hyper-IgM. Nature 361, 539–541 (1993).
  Article CAS PubMed Google Scholar
• Roy, M., Waldschmidt, T., Aruffo, A., Ledbetter, J.A. & Noelle, R.J. The regulation of the expression of gp39, the CD40 ligand, on normal and cloned CD4+ T cells. J. Immunol. 151, 2497–2510 (1993).
  CAS PubMed Google Scholar
• Stout, R.D., Suttles, J., Xu, J., Grewal, I.S. & Flavell, R.A. Impaired T cell-mediated macrophage activation in CD40 ligand-deficient mice. J. Immunol. 156, 8–11 (1996).
  CAS PubMed Google Scholar
• Grewal, I.S. & Flavell, R.A. CD40 and CD154 in cell-mediated immunity. Annu. Rev. Immunol. 16, 111–135 (1998).
  Article CAS PubMed Google Scholar
• Straw, A.D., MacDonald, A.S., Denkers, E.Y. & Pearce, E.J. CD154 plays a central role in regulating dendritic cell activation during infections that induce Th1 or Th2 responses. J. Immunol. 170, 727–734 (2003).
  Article CAS PubMed Google Scholar
• Gennery, A.R. et al. Treatment of CD40 ligand deficiency by hematopoietic stem cell transplantation: a survey of the European experience, 1993-2002. Blood 103, 1152–1157 (2004).
  Article CAS PubMed Google Scholar
• Levy, J. et al. Clinical spectrum of X-linked hyper-IgM syndrome. J. Pediatr. 131, 47–54 (1997).
  Article CAS PubMed Google Scholar
• Hadzic, N. et al. Correction of the hyper-IgM syndrome after liver and bone marrow transplantation. N. Engl. J. Med. 342, 320–324 (2000).
  Article CAS PubMed Google Scholar
• Thomas, C. et al. Brief report: correction of X-linked hyper-IgM syndrome by allogeneic bone marrow transplantation. N. Engl. J. Med. 333, 426–429 (1995).
  Article CAS PubMed Google Scholar
• Brown, M.P. et al. Thymic lymphoproliferative disease after successful correction of CD40 ligand deficiency by gene transfer in mice. Nat. Med. 4, 1253–1260 (1998).
  Article CAS PubMed Google Scholar
• Sacco, M.G. et al. Lymphoid abnormalities in CD40 ligand transgenic mice suggest the need for tight regulation in gene therapy approaches to hyper immunoglobulin M (IgM) syndrome. Cancer Gene Ther. 7, 1299–1306 (2000).
  Article CAS PubMed Google Scholar
• Sullenger, B.A. Targeted genetic repair: an emerging approach to genetic therapy. J. Clin. Invest. 112, 310–311 (2003).
  Article CAS PubMed PubMed Central Google Scholar
• Puttaraju, M., Jamison, S.F., Mansfield, S.G., Garcia-Blanco, M.A. & Mitchell, L.G. Spliceosome-mediated RNA _trans_-splicing as a tool for gene therapy. Nat. Biotechnol. 17, 246–252 (1999).
  Article CAS PubMed Google Scholar
• Garcia-Blanco, M.A., Puttaraju, M., Mansfield, S.G. & Mitchell, L.G. Spliceosome-mediated RNA _trans_-splicing in gene therapy and genomics. Gene Ther. Regul. 1, 141–163 (2000).
  Article CAS Google Scholar
• Liu, X. et al. Partial correction of endogenous DeltaF508 CFTR in human cystic fibrosis airway epithelia by spliceosome-mediated RNA _trans_-splicing. Nat. Biotechnol. 20, 47–52 (2002).
  Article CAS PubMed Google Scholar
• Mansfield, S.G. et al. Repair of CFTR mRNA by spliceosome-mediated RNA _trans_-splicing. Gene Ther. 7, 1885–1895 (2000).
  Article CAS PubMed Google Scholar
• Puttaraju, M., Di Pasquale, J., Baker, C.C., Mitchell, L.G. & Garcia-Blanco, M.A. Messenger RNA repair and restoration of protein function by spliceosome-mediated RNA _trans_-splicing. Mol. Ther. 4, 105–114 (2001).
  Article CAS PubMed Google Scholar
• Chao, H. et al. Phenotype correction of hemophilia A mice by spliceosome-mediated RNA _trans_-splicing. Nat. Med. 9, 1015–1019 (2003).
  Article CAS PubMed Google Scholar
• Garcia-Blanco, M.A. Messenger RNA reprogramming by spliceosome-mediated RNA _trans_-splicing. J. Clin. Invest. 112, 474–480 (2003).
  Article CAS PubMed PubMed Central Google Scholar
• Bonen, L. _Trans_-splicing of pre-mRNA in plants, animals, and protists. FASEB J. 7, 40–46 (1993).
  Article CAS PubMed Google Scholar
• Caudevilla, C. et al. Natural _trans_-splicing in carnitine octanoyltransferase pre-mRNAs in rat liver. Proc. Natl. Acad. Sci. USA 95, 12185–12190 (1998).
  Article CAS PubMed PubMed Central Google Scholar
• Xu, J. et al. Mice deficient for the CD40 ligand. Immunity. 1, 423–431 (1994).
  Article CAS PubMed Google Scholar
• Testi, R., D'Ambrosio, D., De Maria, R. & Santoni, A. The CD69 receptor: a multipurpose cell-surface trigger for hematopoietic cells. Immunol. Today 15, 479–483 (1994).
  Article CAS PubMed Google Scholar
• Kolls, J.K., Beck, J.M., Nelson, S., Summer, W.R. & Shellito, J. Alveolar macrophage release of tumor necrosis factor during murine Pneumocystis carinii pneumonia. Am. J. Respir. Cell Mol. Biol. 8, 370–376 (1993).
  Article CAS PubMed Google Scholar
• Kolls, J.K. et al. IFN-γ and CD8+ T cells restore host defenses against Pneumocystis carinii in mice depleted of CD4+ T cells. J. Immunol. 162, 2890–2894 (1999).
  CAS PubMed Google Scholar
• Logan, A.C., Lutzko, C. & Kohn, D.B. Advances in lentiviral vector design for gene-modification of hematopoietic stem cells. Curr. Opin. Biotechnol. 13, 429–436 (2002).
  Article CAS PubMed Google Scholar
• Baumgartner, R. et al. Evidence for the requirement of T cell costimulation in the pathogenesis of natural Pneumocystis carinii pulmonary infection. Microb. Pathog. 33, 193–201 (2002).
  Article CAS PubMed Google Scholar
• Campbell, K.A. et al. CD40 ligand is required for protective cell-mediated immunity to Leishmania major. Immunity 4, 283–289 (1996).
  Article CAS PubMed Google Scholar
• MacDonald, A.S. et al. Impaired Th2 development and increased mortality during Schistosoma mansoni infection in the absence of CD40/CD154 interaction. J. Immunol. 168, 4643–4649 (2002).
  Article CAS PubMed Google Scholar
• Netea, M.G., Meer, J.W., Verschueren, I. & Kullberg, B.J. CD40/CD40 ligand interactions in the host defense against disseminated Candida albicans infection: the role of macrophage-derived nitric oxide. Eur. J. Immunol. 32, 1455–1463 (2002).
  Article CAS PubMed Google Scholar
• Soong, L. et al. Disruption of CD40-CD40 ligand interactions results in an enhanced susceptibility to Leishmania amazonensis infection. Immunity 4, 263–273 (1996).
  Article CAS PubMed Google Scholar
• Stephens, J., Cosyns, M., Jones, M. & Hayward, A. Liver and bile duct pathology following Cryptosporidium parvum infection of immunodeficient mice. Hepatology 30, 27–35 (1999).
  Article CAS PubMed Google Scholar
• Thomsen, A.R., Nansen, A., Christensen, J.P., Andreasen, S.O. & Marker, O. CD40 ligand is pivotal to efficient control of virus replication in mice infected with lymphocytic choriomeningitis virus. J. Immunol. 161, 4583–4590 (1998).
  CAS PubMed Google Scholar
• Merali, S., Chin, K., Grady, R.W., Weissberger, L. & Clarkson, A.B. Response of rat model of Pneumocystis carinii pneumonia to continuous infusion of deferoxamine. Antimicrob. Agents Chemother. 39, 1442–1444 (1995).
  Article CAS PubMed PubMed Central Google Scholar
• Zheng, M. et al. CD4+ T cell-independent vaccination against Pneumocystis carinii in mice. J. Clin. Invest. 108, 1469–1474 (2001).
  Article CAS PubMed PubMed Central Google Scholar
• Pergolizzi, R.G. et al. _In vivo trans_-splicing of 5′ and 3′ segments of pre-mRNA directed by corresponding DNA sequences delivered by gene transfer. Mol. Ther. 8, 999–1008 (2003).
  Article CAS PubMed Google Scholar
• Armitage, R.J. et al. Molecular and biological characterization of a murine ligand for CD40. Nature 357, 80–82 (1992).
  Article CAS PubMed Google Scholar
• Field, J. et al. Purification of a RAS-responsive adenylyl cyclase complex from Saccharomyces cerevisiae by use of an epitope addition method. Mol. Cell. Biol. 8, 2159–2165 (1988).
  Article CAS PubMed PubMed Central Google Scholar
• Xu, K., Ma, H., McCown, T.J., Verma, I.M. & Kafri, T. Generation of a stable cell line producing high-titer self-inactivating lentiviral vectors. Mol. Ther. 3, 97–104 (2001).
  Article CAS PubMed Google Scholar
• Naldini, L. et al. In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector. Science 272, 263–267 (1996).
  Article CAS PubMed Google Scholar
• Dull, T. et al. A third-generation lentivirus vector with a conditional packaging system. J. Virol. 72, 8463–8471 (1998).
  CAS PubMed PubMed Central Google Scholar
• Szilvassy, S.J., Lansdorp, P.M., Humphries, R.K., Eaves, A.C. & Eaves, C.J. Isolation in a single step of a highly enriched murine hematopoietic stem cell population with competitive long-term repopulating ability. Blood 74, 930–939 (1989).
  CAS PubMed Google Scholar
• Vigna, E. et al. Robust and efficient regulation of transgene expression in vivo by improved tetracycline-dependent lentiviral vectors. Mol. Ther. 5, 252–261 (2002).
  Article CAS PubMed Google Scholar
• Follenzi, A., Ailles, L.E., Bakovic, S., Geuna, M. & Naldini, L. Gene transfer by lentiviral vectors is limited by nuclear translocation and rescued by HIV-1 pol sequences. Nat. Genet. 25, 217–222 (2000).
  Article CAS PubMed Google Scholar
• Kikuchi, T., Worgall, S., Singh, R., Moore, M.A. & Crystal, R.G. Dendritic cells genetically modified to express CD40 ligand and pulsed with antigen can initiate antigen-specific humoral immunity independent of CD4+ T cells. Nat. Med. 6, 1154–1159 (2000).
  Article CAS PubMed Google Scholar