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:
Commentary
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
Descripción
Sumario: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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