Gene therapy and stem cells unite

[rhody]

http://www.bbc.co.uk/news/health-15272081"

Two 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

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

and from the research paper linked in the article:

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.

Rhody...

 

[Ryan_m_b]

One of my greatest hates in the media is how they report stem cell medicine. Research into stem cell therapies, especially iPSCs (which are amazing) has huge promise but not for a long while yet. Unfortunately the media trumps along any incremental improvement in science as a miracle milestone. In my opinion it raises too much false hope in people with dire diseases.

On a less depressing note Virus-free induction of pluripotency and subsequent excision of reprogramming factors by Kaji et al 2009 is one of the best pieces of research I came across this year. In it they outline a remarkable mechanism for using virus free gene therapy to insert pluripotency inducing genes into a cell with the capability to remove the genes afterwards.