Stem cell transplant can rectify infrequent genetic disorder in youths

Posted on 30 December 2012 by Stem Man

Scripps Research Institute scientists have offered new hope for parents whose children suffer from the rare genetic disorder ‘cystinosis’ by showing through an experiment on mice that stem cell transplantation can successfully correct the defect (…)

In the study, the researchers used bone marrow stem cell transplantation to address symptoms of cystinosis in a mouse model (…)

In the new study, the researchers found that transplanted bone marrow stem cells carrying the normal lysosomal cystine transporter gene abundantly engrafted into every tissue of the experimental mice (…)

“The results really surprised and encouraged us. Because the defect is present in every cell of the body, we did not expect a bone marrow stem cell transplant to be so widespread and effective,” says Cherqui.
Cherqui said that adult bone marrow stem cell therapy is particularly well suited as a potential treatment for cystinosis because these cells target all types of tissues.

In addition, stem cells reside in the bone marrow for the duration of a patient”s life, becoming active as needed, a particular benefit for a progressive disease like cystinosis. The study has been published in the journal Blood.

from http://www.newkerala.com/nkfullnews-1-114380.html

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New Stem Cells Technique Offers Hope for Kids With Immune Deficiency

Posted on 3 October 2012 by Stem Man

For children born with immunodeficiencies, researchers may have found a better way for them to get the help they need from stem-cell transplants (…)

Children with primary immunodeficiencies have genetic defects in their immune system that leave them open to infection and other complications. Stem-cell transplants can replace the defective immune system with one derived from healthy donor bone marrow, but without a stem-cell transplant, many of these children might die, the researchers noted in a journal news release.

In order to create space for the donor stem cells and prevent rejection, the patient usually undergoes chemotherapy, radiotherapy or both. This chemoradiotherapy can cause severe liver or lung damage, as well as hair loss and sickness. It may also cause problems with growth, puberty and infertility in later life, according to the news release (…)

With this approach, the 16 children with primary immunodeficiencies in this study, who were too sick for a traditional stem-cell transplant, were able to avoid much of the toxicity caused by chemotherapy (…)

read full article on http://www.ajc.com/health/content/shared-auto/healthnews/bird/630592.html

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Scientists create stem cells to help decipher diseases

Posted on 13 August 2012 by Stem Man

Douglas A. Melton

Scientists have created stem cells from patients suffering from 10 incurable diseases, from Down syndrome to diabetes and Parkinson’s – immortal cells that might one day be turned into repair material for wasting muscles or damaged brains.
The Harvard University-led team has taken skin and bone marrow cells from diseased patients and re-programmed those cells to behave like cells from days-old embryos.

The feat allows scientists for the first time to watch muscular dystrophy and other diseases unfold in a petri dish, “that is, to watch what goes right or wrong,” said Doug Melton, co-director of the Harvard Stem Cell Institute. The cells will also allow researchers to screen new drugs to treat the diseases.
“In these complex genetic diseases, we’re so ignorant at the moment we don’t even know when a patient gets diabetes if they all get it the same way,” Melton said. “There could be 50 different ways to get Type 1 diabetes.” The stem cell lines could help researchers hone in on exactly which mutations are responsible and find “the weak point where you could try to prevent, or treat it.”
“We have good reason to believe that this will make it possible to find new treatments, and eventually drugs, to slow or even stop the course of a number of diseases,” Melton said.
The new cells are “pluripotent” cells that can be coaxed into making any tissue in the human body, and can grow forever.

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Stem cells without genetic defects heralded as breakthrough

Posted on 1 November 2011 by Stem Man

The prospect of treating genetic diseases with corrected stem cells grown from patients’ own bodies has moved closer, after the results of a remarkable experiment.

Scientists have successfully reprogrammed skin tissue from people with a rare form of anaemia to create powerful stem cells, while at the same time rectifying the genetic defect that causes the condition.

The corrected stem cells could be grown into blood precursor cells for therapy. As these would carry a patient’s own DNA, except for the mutation responsible for the illness, they could be transplanted without risk of rejection by the body’s immune system.

Though the research team, from Spain and the United States, has yet to use the cells to treat patients, and several important hurdles still remain, the achievement has been hailed as a significant advance for stem cell research.

It suggests that it should eventually be possible to treat many inherited conditions by making disease-free stem cells from their own bodies. (…)

The cells were infected with a genetically modified virus to correct the gene that causes Fanconi anaemia. These were then reprogrammed into an embryo-like state by modifying further genes, to create versatile master cells known as induced pluripotent stem cells (IPS cells). (…)

Chris Mathew, Professor of Molecular Genetics at King’s College London, said: “This is an important development for families with this rare, inherited blood disorder. The patients have low numbers of blood stem cells in their bone marrow, so there are very few target cells to correct by gene therapy.

“The new research shows that it is possible to reprogramme skin cells from these patients into stem cells in which the genetic defect has been corrected. In future it may become possible to transfer the corrected stem cells back into the patient, but much work remains to be done.”

Read full article on Times Online

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ISRAEL – Embryonic Stem Cell Transplant Causes Benign Tumors in 17 Year-Old Boy

Posted on 9 October 2011 by Stem Man

Researchers using stem cells must work to make their treatments safer after a 17 year-old boy with a rare genetic disease in 2001 was cured with an embryonic stem cell transplant in Moscow, but then developed benign brain and spinal tumors four years later. According to ‘Plos Medicine’ magazine, Israeli doctors removed cancer from the boy, the tumors developed due to a stem cell treatment that he received.

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