Stem-Cell Study Treats Sickle-Cell Anemia in Mice

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Stem-Cell Study Treats Sickle-Cell Anemia in Mice
« on: December 06, 2007, 08:04:09 PM »
Article from Wall-Street Journal:


Stem-Cell Study Treats
Sickle-Cell Anemia in Mice
By GAUTAM NAIK
December 6, 2007 2:06 p.m.

In the latest of a string of stem-cell breakthroughs, scientists have derived embryonic-like stem cells from mature tissue of mice, then used the fresh cells to successfully treat a version of sickle-cell anemia in the animals.

The experiment is an encouraging sign that similar transplant therapies may one day work in humans. It builds on recent advances in which embryonic-like stem cells have been created by reprogramming mature cells in mice and humans -- a promising approach that doesn't require the use of eggs or destruction of embryos, which can be ethically contentious when applied to humans.

People with sickle-cell anemia, which often is fatal, inherit two defective genes for the disease, one from each parent. As a result, there aren't enough healthy red blood cells to carry oxygen throughout the body. Symptoms can include episodes of pain in organs or joints, fatigue and shortness of breath. There are treatments but no cures.

In the latest advance, published Thursday in the online version of the journal Science, researchers used stem cells to treat specially engineered mice that had the same defective genes as someone with sickle-cell anemia. The treatment required several steps.

The scientists first introduced four specific genes into mature skin cells taken from mice. These genes "reprogrammed" the cells and returned them to a more primitive, embryonic-like state. In a lab dish, the cells were then chemically turned into a type of tissue that can generate blood cells in the body.

Next, a normal gene was substituted for the defective "sickle cell" gene in the freshly derived tissue. This step corrected the tissue, which was injected back into the ill mice, where they formed healthy blood cells free from disease.

The mice had previously suffered from rapid breathing, low body weight and low blood counts. After treatment, their condition improved. "It was striking," says Rudolph Jaenisch, whose lab at the Whitehead Institute for Biomedical Research in Cambridge, Mass., did the experiment. "Once you repair it, all the symptoms go away." The paper's lead author is Jacob Hanna, also from Whitehead.

There are limitations to the approach similar to those faced by other recent stem-cell innovations. As part of the reprogramming step, the Whitehead scientists had to use potentially dangerous viruses to introduce the four genes into the mouse's cells. Researchers are now trying to find a benign virus that can do the same trick. While sickle-cell disease is a fairly well-understood malady -- especially the genetic underpinnings -- complex ailments such as Parkinson's will probably be far harder to treat, even in mice.

Nonetheless, the latest piece of science adds to several big advances in recent months in the field of cell reprogramming. Several scientists have shown it is possible to return mature skin cells to an embryonic-like state in both mice and humans. But it will take years -- and a lot more research -- before the transplantation technique can be tried in people.

Write to Gautam Naik at gautam.naik@wsj.com



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Offline §ãJ¡Ð ساجد

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Re: Stem-Cell Study Treats Sickle-Cell Anemia in Mice
« Reply #1 on: December 07, 2007, 04:34:10 PM »
Thanks for the post Bharat.

Narendra posted something similar that said that stem cells can be produced by using one's own cells. I just hope they can reprogram them with the correcting genes and thier transplant would not be rejected.

Well, every single breakthrough counts. Hoping the best for the future!
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Offline KHALIFA

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Re: Stem-Cell Study Treats Sickle-Cell Anemia in Mice
« Reply #2 on: December 07, 2007, 11:44:30 PM »
Dear brother Sajid  really i feel what other thalassimic PpL feel because i had same problem before and i still suffaring from it you don't belive me if i tell you how much i spent since my son Abdulwahab came to this life until i made the STEM-CELL'S transplant to my son ... BUT if any body look back 20 yeras a go he will not going to find out any thing a bout stem cell's transplant .. as you said we are all hoping to find out the genes therapy will be very soon ,, And i remember one day i ask Professor Jhone Porter ( UCH in London ) a bout the gene therapiest he told me we found out the gene  but we don't have the (car) or somthing to cary this gene to the prober place...
                                            khalifa
                                     state of kuwait
                               one for all and all for one
RED_PILOT

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Offline Sharmin

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Re: Stem-Cell Study Treats Sickle-Cell Anemia in Mice
« Reply #3 on: December 08, 2007, 06:08:28 AM »
I am praying that stem cell and gene therapy research will lead to a cure for thalassemia and sickle cell very soon.  I hope that people with many ailments such as cystic fibrosis will be cured soon.  I hope we are close! 
Sharmin

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Offline Andy Battaglia

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Re: Stem-Cell Study Treats Sickle-Cell Anemia in Mice
« Reply #4 on: December 08, 2007, 06:19:28 AM »
The car that Khalifa refers to is called a vector. A vector is a tool used to deliver genetic material into cells. A lentiviral vector is one that is created by taking a virus such as an HIV virus; destroying that part of it which creates HIV; and using this viral “shell” to help get into both dividing and non-dividing cells. This process has cured thalassemia in animals and trials on humans are expected to start in June, 2008. 

From Pat G's Blog at http://blog.myspace.com/index.cfm?fuseaction=blog.view&friendID=148067567&blogID=315621698&Mytoken=AF292071-3EBE-4784-B12B30D5AA4513E979614910

Quote
A Vector has been made to begin the final tests before ordering the Clinical Trial batch.

We are looking at June for a starting of the first patients.

Thanks for your prayers and support.

-Pat g

Andy

All we are saying is give thals a chance.

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Offline §ãJ¡Ð ساجد

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Re: Stem-Cell Study Treats Sickle-Cell Anemia in Mice
« Reply #5 on: December 08, 2007, 08:10:04 AM »
God Bless Pat!

We are with you always!
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