Stem cell-based therapy for α₁-antitrypsin deficiency

Tamir Rashid; David A Lomas

doi:10.1186/scrt95

Stem cell-based therapy for α₁-antitrypsin deficiency

Tamir Rashid, David A Lomas

Centre for Stem Cells & Regenerative Medicine

Research output: Contribution to journal › Article › peer-review

3 Citations (Scopus)

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.

Original language	English
Article number	4
Number of pages	2
Journal	Stem cell research & therapy
Volume	3
Issue number	1
DOIs	https://doi.org/10.1186/scrt95
Publication status	Published - 9 Feb 2012

Keywords

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

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10.1186/scrt95

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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.",

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AB - 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.

KW - Alleles

KW - Humans

KW - Point Mutation

KW - Stem Cell Transplantation

KW - Stem Cells

KW - Transplantation, Autologous

KW - alpha 1-Antitrypsin Deficiency

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Stem cell-based therapy for α₁-antitrypsin deficiency

Abstract

Keywords

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