Augmentation of blood platelet levels by intratracheal administration of an adenovirus vector encoding human thrombopoietin cDNA

Nat Biotechnol. 1997 Jun;15(6):570-3. doi: 10.1038/nbt0697-570.

Abstract

This study was designed to evaluate the hypothesis that administration of a replication-deficient, recombinant adenovirus vector to the epithelial surface of the respiratory tract can be used to deliver a recombinant protein to the systemic circulation in sufficient quantities to evoke a systemic response appropriate to the recombinant protein. We administered AdCMV.TPO-an adenovirus vector containing an expression cassette coding for the human thrombopoietin (TPO) cDNA-to the respiratory epithelium of immunocompetent Balb/c mice. Over the following week, serum human TPO levels were elevated, platelet levels increased more than sixfold, and megakaryocytosis was evident in bone marrow. This strategy may be a useful approach to the nonparenteral administration of a variety of therapeutic recombinant proteins, such as those relevant to clotting, endocrine function, and bone-marrow function.

Publication types

  • Research Support, Non-U.S. Gov't
  • Research Support, U.S. Gov't, P.H.S.

MeSH terms

  • Adenoviridae
  • Animals
  • Bone Marrow Cells
  • Cell Line
  • Cytomegalovirus
  • DNA, Complementary
  • Gene Transfer Techniques*
  • Genetic Therapy
  • Genetic Vectors
  • Hematopoietic Stem Cells / cytology
  • Humans
  • Megakaryocytes / cytology
  • Mice
  • Mice, Inbred BALB C
  • Mutagenesis, Insertional
  • Platelet Count*
  • Recombinant Proteins / biosynthesis
  • Recombinant Proteins / blood
  • Spleen / cytology
  • Thrombopoietin / biosynthesis
  • Thrombopoietin / blood
  • Thrombopoietin / genetics*

Substances

  • DNA, Complementary
  • Recombinant Proteins
  • Thrombopoietin