Targeted gene correction of α1-antitrypsin deficiency in induced pluripotent stem cells

@inproceedings{Yusa2011TargetedGC,
  title={Targeted gene correction of α1-antitrypsin deficiency in induced pluripotent stem cells},
  author={Kosuke Yusa and Sheikh Tamir Rashid and Helene Strick-Marchand and Ignacio Varela and P Q Liu and David E. Paschon and Elena Miranda and Adriana del V. Ord{\'o}{\~n}ez and N. R.-F. Hannan and Foad Jafari Rouhani and Sylvie Darche and Graeme J Alexander and Stefan J Marciniak and Noemi Fusaki and Mamoru Hasegawa and Michael C. Holmes and James P Di Santo and David A. Lomas and Allan Bradley and Ludovic Vallier},
  booktitle={Nature},
  year={2011}
}
Human induced pluripotent stem cells (iPSCs) represent a unique opportunity for regenerative medicine because they offer the prospect of generating unlimited quantities of cells for autologous transplantation, with potential application in treatments for a broad range of disorders. However, the use of human iPSCs in the context of genetically inherited human disease will require the correction of disease-causing mutations in a manner that is fully compatible with clinical applications. The… CONTINUE READING
Highly Influential
This paper has highly influenced a number of papers. REVIEW HIGHLY INFLUENTIAL CITATIONS