| First Author | Spitzer D | Year | 2006 |
| Journal | J Immunol | Volume | 177 |
| Issue | 8 | Pages | 4953-6 |
| PubMed ID | 17015675 | Mgi Jnum | J:139301 |
| Mgi Id | MGI:3807726 | Doi | 10.4049/jimmunol.177.8.4953 |
| Citation | Spitzer D, et al. (2006) Cutting edge: treatment of complement regulatory protein deficiency by retroviral in vivo gene therapy. J Immunol 177(8):4953-6 |
| abstractText | Gene therapy is an attractive means to replace a deficient or defective protein. Using a murine retroviral vector, we provide an example of reconstituting a C regulator by neonatal in vivo gene transfer. The fusion gene containing the mouse C receptor 1-related gene/protein y (Crry) and a single chain Ab fragment with specificity for mouse glycophorin A was placed under transcriptional control of a liver-specific promoter. Shortly after birth, Crry KO mice were injected with the retroviral vectors. Protein expression progressively increased over the next 6-8 wk after which an equilibrium was established. Coating levels on RBCs were obtained that inhibited C activation similar to wild-type cells and remained constant for > 1 year. Thus, gene therapy with targeted regulators represents a treatment option to provide a long-term and sustained protein supply for the site-specific blockade of undesirable complement activation. |
