andTwo of the holy grails of medicine - stem cell technology and precision gene therapy - have been united for the first time in humans, say scientists.
and from the research paper linked in the article:The research group took a skin cell from a patient and converted it to a stem cell.
A molecular scalpel was used to cut out the single mutation and insert the right letter - correcting the genetic fault.
The stem cells were then turned into liver cells. One of the lead researchers, Prof David Lomas, said: "They functioned beautifully with normal secretion and function
Rhody...Genetic correction of human iPSCs restored the structure and function of A1AT in subsequently derived liver cells in vitro and in vivo. This approach is significantly more efficient than any other gene-targeting technology that is currently available and crucially prevents contamination of the host genome with residual non-human sequences. Our results provide the first proof of principle, to our knowledge, for the potential of combining human iPSCs with genetic correction to generate clinically relevant cells for autologous cell-based therapies.
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