Stable differentiation and clonality of murine long-term hematopoiesis after extended reduced-intensity selection for MGMT P140K transgene expression

Blood. 2007 Sep 15;110(6):1779-87. doi: 10.1182/blood-2006-11-053710. Epub 2007 May 11.

Abstract

Efficient in vivo selection increases survival of gene-corrected hematopoietic stem cells (HSCs) and protects hematopoiesis, even if initial gene transfer efficiency is low. Moreover, selection of a limited number of transduced HSCs lowers the number of cell clones at risk of gene activation by insertional mutagenesis. However, a limited clonal repertoire greatly increases the proliferation stress of each individual clone. Therefore, understanding the impact of in vivo selection on proliferation and lineage differentiation of stem-cell clones is essential for its clinical use. We established minimal cell and drug dosage requirements for selection of P140K mutant O6-methylguanine-DNA-methyltransferase (MGMT P140K)-expressing HSCs and monitored their differentiation potential and clonality under long-term selective stress. Up to 17 administrations of O6-benzylguanine (O6-BG) and 1,3-bis(2-chloroethyl)-1-nitroso-urea (BCNU) did not impair long-term differentiation and proliferation of MGMT P140K-expressing stem-cell clones in mice that underwent serial transplantation and did not lead to clonal exhaustion. Interestingly, not all gene-modified hematopoietic repopulating cell clones were efficiently selectable. Our studies demonstrate that the normal function of murine hematopoietic stem and progenitor cells is not compromised by reduced-intensity long-term in vivo selection, thus underscoring the potential value of MGMT P140K selection for clinical gene therapy.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Antineoplastic Agents / pharmacology
  • Antineoplastic Agents, Alkylating / pharmacology
  • Bone Marrow Cells / drug effects
  • Bone Marrow Cells / enzymology*
  • Bone Marrow Transplantation
  • Carmustine / pharmacology
  • Cell Differentiation*
  • Genetic Therapy / methods
  • Guanine / analogs & derivatives
  • Guanine / pharmacology
  • Hematopoiesis / drug effects
  • Hematopoiesis / physiology*
  • Mice
  • Mice, Inbred C57BL
  • Mice, Transgenic
  • Myeloid Cells / drug effects
  • Myeloid Cells / metabolism
  • O(6)-Methylguanine-DNA Methyltransferase / genetics*
  • O(6)-Methylguanine-DNA Methyltransferase / metabolism
  • Stem Cells / drug effects
  • Stem Cells / metabolism*
  • Terminal Repeat Sequences / physiology
  • Transduction, Genetic
  • Transplantation, Homologous

Substances

  • Antineoplastic Agents
  • Antineoplastic Agents, Alkylating
  • O(6)-benzylguanine
  • Guanine
  • O(6)-Methylguanine-DNA Methyltransferase
  • Carmustine