Reduction in levels of the cyclin-dependent kinase inhibitor p27(kip-1) coupled with transforming growth factor beta neutralization induces cell-cycle entry and increases retroviral transduction of primitive human hematopoietic cells

Proc Natl Acad Sci U S A. 1998 Oct 27;95(22):13006-11. doi: 10.1073/pnas.95.22.13006.

Abstract

Successful gene therapy depends on stable transduction of hematopoietic stem cells. Target cells must cycle to allow integration of Moloney-based retroviral vectors, yet hematopoietic stem cells are quiescent. Cells can be held in quiescence by intracellular cyclin-dependent kinase inhibitors. The cyclin-dependent kinase inhibitor p15(INK4B) blocks association of cyclin-dependent kinase (CDK)4/cyclin D and p27(kip-1) blocks activity of CDK2/cyclin A and CDK2/cyclin E, complexes that are mandatory for cell-cycle progression. Antibody neutralization of beta transforming growth factor (TGFbeta) in serum-free medium decreased levels of p15(INK4B) and increased colony formation and retroviral-mediated transduction of primary human CD34(+) cells. Although TGFbeta neutralization increased colony formation from more primitive, noncycling hematopoietic progenitors, no increase in M-phase-dependent, retroviral-mediated transduction was observed. Transduction of the primitive cells was augmented by culture in the presence of antisense oligonucleotides to p27(kip-1) coupled with TGFbeta-neutralizing antibodies. The transduced cells engrafted immune-deficient mice with no alteration in human hematopoietic lineage development. We conclude that neutralization of TGFbeta, plus reduction in levels of the cyclin-dependent kinase inhibitor p27, allows transduction of primitive and quiescent hematopoietic progenitor populations.

Publication types

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

MeSH terms

  • Animals
  • Antibodies / pharmacology
  • Antigens, CD / analysis
  • Antigens, CD34 / analysis
  • Bone Marrow Cells / cytology
  • Cell Cycle / drug effects
  • Cell Cycle / physiology*
  • Cell Cycle Proteins*
  • Clone Cells
  • Colony-Forming Units Assay
  • Cyclin-Dependent Kinase Inhibitor p27
  • Cyclin-Dependent Kinases / antagonists & inhibitors*
  • Genetic Vectors
  • Hematopoietic Stem Cells / cytology*
  • Hematopoietic Stem Cells / drug effects
  • Hematopoietic Stem Cells / physiology*
  • Humans
  • Immunomagnetic Separation
  • Mice
  • Mice, Nude
  • Mice, SCID
  • Microtubule-Associated Proteins / antagonists & inhibitors
  • Microtubule-Associated Proteins / metabolism*
  • Moloney murine leukemia virus
  • Oligonucleotides, Antisense / pharmacology
  • T-Lymphocytes / cytology
  • Thionucleotides
  • Transduction, Genetic
  • Transforming Growth Factor beta / antagonists & inhibitors
  • Transforming Growth Factor beta / immunology
  • Transforming Growth Factor beta / pharmacology*
  • Tumor Suppressor Proteins*

Substances

  • Antibodies
  • Antigens, CD
  • Antigens, CD34
  • Cdkn1b protein, mouse
  • Cell Cycle Proteins
  • Microtubule-Associated Proteins
  • Oligonucleotides, Antisense
  • Thionucleotides
  • Transforming Growth Factor beta
  • Tumor Suppressor Proteins
  • Cyclin-Dependent Kinase Inhibitor p27
  • Cyclin-Dependent Kinases