Stem cell-based therapy for α₁-antitrypsin deficiency

Tamir Rashid, David A Lomas

Research output: Contribution to journalArticlepeer-review

3 Citations (Scopus)


Human induced pluripotent stem cells offer the possibility of generating unlimited quantities of cells for autologous transplantation. By correcting the genetic defect underlying Z-allele α₁-antitrypsin deficiency, we recently provided the first proof of principle for application of human induced pluripotent stem cells in the treatment of inherited genetic disorders. Several important safety concerns will need to be addressed before this can be translated into clinical practice.
Original languageEnglish
Article number4
Number of pages2
JournalStem cell research & therapy
Issue number1
Publication statusPublished - 9 Feb 2012


  • Alleles
  • Humans
  • Point Mutation
  • Stem Cell Transplantation
  • Stem Cells
  • Transplantation, Autologous
  • alpha 1-Antitrypsin Deficiency


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