Efficient Correction of Hemoglobinopathy-Causing Mutations by Homologous Recombination in Integration-Free Patient iPSCs

Cell Res. 2011 Dec;21(12):1740-4. doi: 10.1038/cr.2011.186. Epub 2011 Nov 22.

Publication types

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

MeSH terms

  • Adenoviridae / genetics
  • Fibroblasts / cytology
  • Fibroblasts / metabolism
  • Genetic Vectors / metabolism
  • Hemoglobinopathies / genetics*
  • Hemoglobinopathies / therapy
  • Hemoglobins / genetics
  • Homologous Recombination*
  • Humans
  • Induced Pluripotent Stem Cells / cytology*
  • Induced Pluripotent Stem Cells / transplantation
  • Mutation*
  • Targeted Gene Repair*

Substances

  • Hemoglobins