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Stem cell-based therapy for α(1)-antitrypsin deficiency

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 α(1)-antitrypsin deficiency, we recently provided the first proof of principle for application of human induced...

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Detalles Bibliográficos
Autores principales: Rashid, S Tamir, Lomas, David A
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2012
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3340548/
https://www.ncbi.nlm.nih.gov/pubmed/22340671
http://dx.doi.org/10.1186/scrt95
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author Rashid, S Tamir
Lomas, David A
author_facet Rashid, S Tamir
Lomas, David A
author_sort Rashid, S Tamir
collection PubMed
description 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 α(1)-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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spelling pubmed-33405482013-02-09 Stem cell-based therapy for α(1)-antitrypsin deficiency Rashid, S Tamir Lomas, David A Stem Cell Res Ther Commentary 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 α(1)-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. BioMed Central 2012-02-09 /pmc/articles/PMC3340548/ /pubmed/22340671 http://dx.doi.org/10.1186/scrt95 Text en Copyright ©2012 BioMed Central Ltd
spellingShingle Commentary
Rashid, S Tamir
Lomas, David A
Stem cell-based therapy for α(1)-antitrypsin deficiency
title Stem cell-based therapy for α(1)-antitrypsin deficiency
title_full Stem cell-based therapy for α(1)-antitrypsin deficiency
title_fullStr Stem cell-based therapy for α(1)-antitrypsin deficiency
title_full_unstemmed Stem cell-based therapy for α(1)-antitrypsin deficiency
title_short Stem cell-based therapy for α(1)-antitrypsin deficiency
title_sort stem cell-based therapy for α(1)-antitrypsin deficiency
topic Commentary
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3340548/
https://www.ncbi.nlm.nih.gov/pubmed/22340671
http://dx.doi.org/10.1186/scrt95
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