Stem cell-based therapy for α1-antitrypsin deficiency

被引：0
|
作者：
S Tamir Rashid
David A Lomas
机构：
[1] University of Cambridge,Department of Medicine
[2] Cambridge Institute for Medical Research,Laboratory for Regenerative Medicine
[3] University of Cambridge,undefined
来源：
Stem Cell Research & Therapy | / 3卷
关键词：
Pluripotent Stem Cell; iPSC; Zinc Finger Nuclease; Human iPSC; Human Induce Pluripotent Stem Cell;
D O I：
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摘要：
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.
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