title: Stem cell-based therapy for α₁-antitrypsin deficiency.
abstract: 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.
authors.affiliation: Department of Medicine, University of Cambridge, Cambridge Institute for Medical Research, Wellcome Trust/MRC Building, Hills Road, Cambridge CB2 0XY, UK.
authors.forename: S Tamir
authors.initials: ST
authors.lastname: Rashid
authors.forename: David A
authors.initials: DA
authors.lastname: Lomas
mesh_terms: D000483:Alleles; D006801:Humans; D017354:Point Mutation; D033581:Stem Cell Transplantation; D013234:Stem Cells / Q000166:cytology*; D014182:Transplantation, Autologous; D019896:alpha 1-Antitrypsin Deficiency / Q000235:genetics / Q000628:therapy*