Review
Gene Therapy (2008) 15, 82–88; doi:10.1038/sj.gt.3303061; published online 15 November 2007
Scalable human ES culture for therapeutic use: propagation, differentiation, genetic modification and regulatory issues
M Rao1
1Stem Cells and Regenerative Medicine, Invitrogen Corporation, Carlsbad, CA, USA
Correspondence: Dr M Rao, Stem Cells and Regenerative Medicine, Invitrogen Corporation, 5781 Van Allen Way, Carlsbad, CA 92008, USA. E-mail: mahendra.rao@invitrogen.com
Received 8 September 2007; Revised 8 October 2007; Accepted 10 October 2007; Published online 15 November 2007.
Abstract
Embryonic stem cells unlike most adult stem cell populations can replicate indefinitely while preserving genetic, epigenetic, mitochondrial and functional profiles. ESCs are therefore an excellent candidate cell type for providing a bank of cells for allogenic therapy and for introducing targeted genetic modifications for therapeutic intervention. This ability of prolonged self-renewal of stem cells and the unique advantages that this offers for gene therapy, discovery efforts, cell replacement, personalized medicine and other more direct applications requires the resolution of several important manufacturing, gene targeting and regulatory issues. In this review, we assess some of the advance made in developing scalable culture systems, improvement in vector design and gene insertion technology and the changing regulatory landscape.
Keywords:
Good Manufacturing Practice (GMP), ESC-like, screening, clinical grade
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