Skip to main content

Thank you for visiting nature.com. You are using a browser version with limited support for CSS. To obtain the best experience, we recommend you use a more up to date browser (or turn off compatibility mode in Internet Explorer). In the meantime, to ensure continued support, we are displaying the site without styles and JavaScript.

  • News & Views
  • Published:

Coaxing coagulation with RNA and cell fragments

Two new therapeutic strategies show promise in mouse models of hemophilia. One uses an RNA repair approach relying on trans-splicing to patch a genetic defect in a mouse model. Another aims at microparticles, cell fragments that seem to promote coagulation (pages 1015–1019 and 1020–1025).

This is a preview of subscription content, access via your institution

Access options

Buy this article

Prices may be subject to local taxes which are calculated during checkout

Figure 1: The coagulation cascade.
Figure 2: RNA repair by spliceosome-mediated RNA trans-splicing.

References

  1. High, K.A. Gene transfer as an approach to treating hemophilia. Circ. Res. 88, 137–144 (2001).

    Article  CAS  Google Scholar 

  2. Kay, M.A. et al. Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector. Nat. Genet. 24, 257–261 (2000).

    Article  CAS  Google Scholar 

  3. Manno, C.S. et al. AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B. Blood 101, 2963–2972 (2003).

    Article  CAS  Google Scholar 

  4. Roth, D.A., Tawa, N.E., O'Brien, J.M., Treco, D.A. & Selden, R.F. Nonviral transfer of the gene encoding coagulation factor VIII in patients with severe hemophilia A. N. Engl. J. Med. 344, 1735–1742 (2001).

    Article  CAS  Google Scholar 

  5. Hrachovinova, I. et al. P-selectin/PSGL-1 interaction generates microparticles that correct hemostasis in a murine model of hemophilia A. Nat. Med. (2003).

  6. Chao, H. et al. Phenotype correction of hemophilia A mice by spliceosome-mediated RNA trans-splicing. Nat. Med. (2003).

  7. Falati, S. et al. Accumulation of tissue factor into developing thrombi in vivo is dependent upon microparticle P-selectin glycoprotein ligand 1 and platelet P-selectin. J. Exp. Med. 197, 1585–1598 (2003).

    Article  CAS  Google Scholar 

  8. Myers, D. et al. New and effective treatment of experimentally induced venous thrombosis with anti-inflammatory rPSGL-Ig. Thromb. Haemost. 87, 374–382 (2002).

    Article  CAS  Google Scholar 

  9. Bi, L. et al. Targeted disruption of the mouse factor VIII gene: a model for hemophilia A. Nat. Genet. 10, 119–121 (1995).

    Article  CAS  Google Scholar 

Download references

Author information

Authors and Affiliations

Authors

Rights and permissions

Reprints and permissions

About this article

Cite this article

High, K. Coaxing coagulation with RNA and cell fragments. Nat Med 9, 991–992 (2003). https://doi.org/10.1038/nm0803-991

Download citation

  • Issue Date:

  • DOI: https://doi.org/10.1038/nm0803-991

Search

Quick links

Nature Briefing

Sign up for the Nature Briefing newsletter — what matters in science, free to your inbox daily.

Get the most important science stories of the day, free in your inbox. Sign up for Nature Briefing