Free Republic
Browse · Search
Topics · Post Article

Skip to comments.

Gene therapy and stem cells unite
BBC ^ | October 12, 2011 | James Gallagher

Posted on 10/13/2011 6:30:34 PM PDT by CutePuppy

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.

It means patients with a genetic disease could, one day, be treated with their own cells.

A study in Nature corrected a mutation in stem cells made from a patient with a liver disease.

Researchers said this was a "critical step" towards devising treatments, but safety tests were still needed.

At the moment, stem cells created from a patient with a genetic illness cannot be used to cure the disease as those cells would also contain the corrupted genetic code.

Scientists, at the Wellcome Trust Sanger Institute and the University of Cambridge, were working on cirrhotic liver disease.

It is caused by a change to a single pair of letters, out of the six billion which make up the genetic code.

As a result, a protein which protects the body from damage, antitrypsin, cannot escape from the liver where it is made.

..... < snip >

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".

..... < snip >

Further animal studies and human clinical trials would be needed before any treatment as "the key thing is safety".


(Excerpt) Read more at ...

TOPICS: Culture/Society; News/Current Events; United Kingdom
KEYWORDS: cancer; cirrhosis; genes; genetherapy; genetics; liver; medicine; personalizedmedicine; stemcells
Professor David Lomas, one of the lead researchers, described the procedure as "ridiculously hard" but with "enormous potential".
1 posted on 10/13/2011 6:30:39 PM PDT by CutePuppy
[ Post Reply | Private Reply | View Replies]

To: neverdem; decimon
This is another big step in the "personalized medicine."

From related article: Human 'cloning' makes embryonic stem cells - BBC, by James Gallagher, 2011 October 05

A form of cloning has been used to create personalised embryonic stem cells in humans, say researchers.

Genetic material was taken from an adult skin cell and transferred into a human egg. This was grown to produce an early embryo.

Stem cells have huge potential in medicine as they can transform into any other cell type in the body.

However, the stem cells formed contained chromosomes from both the adult and the egg cells.

..... < snip >

The lead researcher at the New York Stem Cell Foundation Laboratory, Dr Dieter Egli, said ... other "groups had tried before, but failed".

Writing in the journal Nature, he said his group had also failed using traditional techniques.

When they removed the genetic material from the egg and replaced it with the chromosomes from a skin cell, the egg divided but failed to go past the 6-12 cell stage.

However, when they left the egg's own genetic material in place and added the skin chromosomes, the egg developed. It reached the blastocyst stage, which can contain up to 100 cells and is the usual source of embryonic stem cells.

..... < snip >

Dr Egli told the BBC: "The cells we have made are not yet for therapeutic use. There is clearly more work to be done, this is early days. ... "While this approach does not in itself provide a solution, it takes us a step closer to understanding where the problems lie."

No embryos required

Recently a different route to stem cells has been used. Instead of using an egg, a chemical bath "reprogrammes" an adult cell into a stem cell. ..... < snip >

2 posted on 10/13/2011 6:34:04 PM PDT by CutePuppy (If you don't ask the right questions you may not get the right answers)
[ Post Reply | Private Reply | To 1 | View Replies]

To: CutePuppy

2 Holy Grails = BIG Pandora’s Box!
just saying

3 posted on 10/13/2011 6:40:52 PM PDT by acapesket
[ Post Reply | Private Reply | To 2 | View Replies]

To: CutePuppy; neverdem; DvdMom; grey_whiskers; Ladysmith; Roos_Girl; Silentgypsy; conservative cat; ...


Thanks, Cute.

4 posted on 10/13/2011 6:44:19 PM PDT by decimon
[ Post Reply | Private Reply | To 2 | View Replies]

To: CutePuppy
From related article: Human 'cloning' makes embryonic stem cells

Thanks for reminding me about that story. I saw it last week at ScienceNOW, Science's free website. Alpha 1-antitrypsin deficiency is the name of the disease for this gene therapy/induced pluripotent stem cell stoty. I just posted it. It has the abstract from Nature.

Spell-Checked Stem Cells Show Promise Against Liver Disease

5 posted on 10/13/2011 7:31:19 PM PDT by neverdem (Xin loi minh oi)
[ Post Reply | Private Reply | To 2 | View Replies]

To: decimon; neverdem; martin_fierro; AdmSmith; AnonymousConservative; Berosus; bigheadfred; ...

Thanks CutePuppy.
...cirrhotic liver disease... is caused by a change to a single pair of letters, out of the six billion which make up the genetic code.

6 posted on 10/14/2011 4:07:53 AM PDT by SunkenCiv (It's never a bad time to FReep this link --
[ Post Reply | Private Reply | View Replies]

Disclaimer: Opinions posted on Free Republic are those of the individual posters and do not necessarily represent the opinion of Free Republic or its management. All materials posted herein are protected by copyright law and the exemption for fair use of copyrighted works.

Free Republic
Browse · Search
Topics · Post Article

FreeRepublic, LLC, PO BOX 9771, FRESNO, CA 93794 is powered by software copyright 2000-2008 John Robinson