Tag Archive for 'Genetic disorder'

Stem cell transplant can rectify infrequent genetic disorder in youths

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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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Stem cells without genetic defects heralded as breakthrough

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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

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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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Scientists create stem cells to help decipher diseases

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Douglas A. Melton

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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USA – Stem Cells, Possible to Reproduce Diseases in Laboratory

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Organica
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An experiment successfully performed by researchers at the University of Wisconsin-Madison and the University of Missouri in Colombia, described in Nature magazine shows that cells removed from a patient’s skin and transformed into cells similar to embryonic stem cells have become a laboratory model for diseases and can be observed in real time and studied to find new cures. The researchers recreated spinal muscular atrophy (SMA) pluripotent stem cells removed from the skin of a child affected by the neurodegenerative genetic disease. In the laboratory, the cells behaved exactly as they do in a human being, giving the researchers an opportunity to observe the progression of the disease. This is an entirely new opportunity, made possible by a discovery by a Japanese research group, which less than a year ago was able to reprogram adult embryonic-like stem cells.

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Stem Cells used to Grow Bone in Boy’s Jowl

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Cincinnati Children’s Hospital Medical Center, known for “miracle” surgeries, has completed a medically historic procedure where doctors used stem cells taken from the fat tissue of a 14-year-old boy and combined them with growth protein and donor tissue to grow viable cheek bones in the teen.

Brad Guilkey, 15, suffers from a rare kind of genetic disorder known as Treacher Collins Syndrome, where the bones and other tissues are prevented to develop in the face (…)

The medical team implanted cadaver bone into Brad’s face in May, and then they injected his own stem cells into the donor bone to fill in the gaps (…)

from http://www.efitnessnow.com/news/2009/10/13/doctors-use-stem-cells-to-grow-bone-in-boys-cheek/

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