Functional Repair of CFTR by CRISPR in Intestinal Stem Cells

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enniob

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Staff member
Promising results from a gene therapy study late last year by Hans Clevers, a Dutch stem cell researcher, showed that a technique called CRISPR can be used to correct the CFTR gene defect associated with cystic fibrosis in human stem cells. Using cultured intestinal stem cells, they were able to correct the defect. The researchers then grew the cells into intestinal “organoids,” or miniature guts, and showed that they functioned normally. In this case, about half of clonal organoids underwent the proper genetic correction, Clevers said.

This is a really interesting study that could see "true" gene therapy be applied via stem cells to the human body. There is still a lot of research to be done on this method, but the technology of correcting gene defects and sustainably incorporating them into cells and organs is actually here and has been demonstrated to work in mice and human stem cells.

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Image taken from Cell publication
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enniob

Administrator
Staff member
This particular study is localized to small intestine. They used small intestine because of the abundance of stem cells. So its a great place to provide proof of concept for this technique. However, This technique can be applied to many other organs and in time potentially the whole body (but that would be a fair way away). I think organ localized gene therapy approaches like this one for genetic diseases may not be so far away as some may think. Watch this 4min video from Hans Clevers that describes the approach and why small intestine is used.

[video=vimeo;26262714]http://vimeo.com/26262714[/video]
 
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Mallika

New member
Would that mean, this only works for people who's stem cells(umblical chord) is stored during their birth?
Any information on this if you know..
Anyway Amazing blog
 
E

enniob

Administrator
Staff member
Umbilical cord is simply a very good source of good quality stem cells. If these are not available, its still possible to use stem cells from bone marrow (and other sources), but there are increased challenges and risks involved as they are harder to differentiate into cells like lung epithelial cells. Lungs are not a good source of stem cells unfortunately. So to answer your question: Yes its possible without umbilical cord stem cells.

There are two types of stem cells: Embryonic stem cells (from embrio) and adults stem cells which are derived from the umbilical cord, placenta or from blood, bone marrow, skin, small intestine and other tissues. Embryonic stem cells have the capacity to develop into every type of tissue found in an adult. Adult stem cells have been found in many organs and tissues of the body, but, once removed from the body, they have a difficult time dividing, which makes generating large quantities of them quite challenging.

For CF, we ideally want to use stem cell therapy to grow lung cells (epithelial cells) that have a restored CFTR gene and protein. Even better, we could intervene at the embryonic stage and fix the CFTR gene before the fetus develops...but there are huge ethical issues with that method.

- Chris
 
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