Cutting edge: Treatment of complement regulatory protein deficiency by retroviral in vivo gene therapy

Dirk Spitzer, Xiabo Wu, Xiucui Ma, Lingfei Xu, Katherine P. Ponder, John P. Atkinson

Research output: Contribution to journalArticle

10 Scopus citations

Abstract

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.

Original languageEnglish
Pages (from-to)4953-4956
Number of pages4
JournalJournal of Immunology
Volume177
Issue number8
DOIs
StatePublished - Oct 15 2006

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