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

ABSTRACT: 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 methods currently available, such as homologous recombination, lack the necessary efficiency and also leave residual sequences in the targeted genome. Therefore, the development of new approaches to edit the mammalian genome is a prerequisite to delivering the clinical promise of human iPSCs. Here we sho

SUBMITTER: Yusa K 

PROVIDER: S-EPMC3198846 | biostudies-literature | 2011 Oct

REPOSITORIES: biostudies-literature