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Tuesday September 22, 2009
Sangamo (SGMO): Progress in adult stem calls?
In The Medical Technology Stock Letter, the biotech specialist adds, "Part of the excitement about stem cells is the concept that one day scientists will be able to create 'personalized' stem cell types for individual therapy. This could be the break needed to realize this potential." "Stem cells differ from other cell types in two fundamental ways. First, they are unspecialized cells capable of renewing themselves through cell division. "Second, under certain conditions, they can be induced to become tissue- or organ-specific cells with special functions. 
"Stem cells have the potential to develop into the body's many different cell types and in many tissues they serve a repair function, differentiating and replacing damaged cells. iPSCs are adult cells that have been genetically reprogrammed to an embryonic stem cell-like state. "Sangamo BioSciences and scientists from The Whitehead Institute have used zinc finger nucleases (ZFNs) to efficiently and precisely modify the genomes of human embryonic stem cells (hESCs) and induced pluripotent stem cells (hiPSCs). "An advantage of iPSCs is that they can be derived from the adult cells of a person and will possess the same genetic background as that individual. Human ESCs and iPSCs are useful tools in drug discovery and should have potential as human therapeutics as the technology develops. "Specifically, the scientists hope to use these cells therapeutically in transplantation medicine and other regenerative applications. "The research was described in a paper which appeared in the scientific journal Nature Biotechnology. "In the article published today, Dr. Jaenisch and his team demonstrated the broad applicability of gene editing applications enabled by ZFNs in hESCs and hiPSCs. "In one example, they generated hESCs that carried a reporter gene. This gave researchers a visual method to clearly identify undifferentiated cells from those that had differentiated to their final cell type. "In another example they inserted a new gene into a specific site in the genome of hESCs and demonstrated that ZFNs can be used to generate reporter cells in non-expressed genes in the genomes of hESCs and iPSCs, tools that will be useful in the study of cell fate and differentiation protocols. "In our view, this is huge news as it opens up a very large opportunity for new therapeutics and one would guess more partnerships. We expect many of the stem cell developers to license the technology. "We are very excited about the company and believe SGMO is uniquely positioned to create a powerhouse drug discovery company. We believe that if any of their therapeutic programs catch the attention of Wall Street, the stock will be driven much higher." 
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