TALENs™: Genome Customization Tools for Precise Gene Editing of Human Stem Cells
Thursday, 28 June 2012 at 16:15
Human stem cells, especially patient-derived induced pluripotent stem cells, represent power-full candidates for improvements to current drug screening approaches and for the development of novel regenerative medicines. In such applications however, genome customization is needed for genetic-abnormalities repair, as for more efficient and homogenous cell differentiation to a complete mature stage. Targeted gene editing is entering an exciting new era with the recent advent of TAL Effector Nucleases (TALENs™). They are able to bind to any gene and introduce a specific DNA double strand break at any position, enabling genetic modification in any cell type. Furthermore, as TALENs™ hits are found every 2 to 3 nucleotides, TALENs™ offer advances in both flexibility of design and universality of use for gene modification. Here is shown use of TALEN™ in human embryonic and induced pluripotent stem cells, for targeted gene integration applications. Combined with a robust feeder free culture system, DEF –CSTM, TALEN™ allows fusion of the GFP to the beta actin. This is an efficient, robust and precise approach that opens the door to an “on request” stem cells engineering.
Registration
Wednesday, 27 June 2012 at 08:00
Stem Cell Therapies: To the Clinic Through the Clinic
Wednesday, 27 June 2012 at 14:15
Stem cell-based regenerative medicine shows much promise. The challenge is to develop safe and cost-effective therapies. Existing biotech business models are sorely stretched when applied to cellular therapies. Market models based on close clinician-company partnerships are likely to bring the earliest successes.
