“Latest Stem Cells News”

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/

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

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

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

Scientists are one step closer to creating a gene therapy/stem cell combination to combat genetic diseases. With work, this research may lead to not only curing the disease, but also repairing the damage left behind.

While gene therapy is a burgeoning field that has shown great results in treating genetic disorders, many of those diseases leave behind heavily damaged tissue that the body is unable to repair. So even if the disease is completely eradicated, quality of life may not necessarily improve, and without help, health can still continue to deteriorate.

Since stem cell research began, there has been a hope that use of those cells may help alleviate some of the trauma left behind by genetic diseases. While the theory has been shown to work in mice, this is the first time a human cell line has confirmed that it is possible the therapy will work for humans as well.

Researchers at the Salk Institute for Biological Studies in La Jolla, California, chose to focus on Fanconi anemia (FA), a genetic disorder that is characterized by short stature, bone marrow failure, irregularities in blood cells that can lead to clotting, and an increased risk of leukemia. Even after gene therapy and “bone marrow transplants to correct the hematological [blood] problems, patients remain at high risk of developing cancer and other serious health conditions.”

The Nature-published technique employed by Juan-Carlos Izpisúa Belmonte’s team was two-fold. First, they collected hair and skin cells from those suffering from FA. These types of cells are known as somatic cells, and can be used to create induced pluripotent stem (iPS) cells. Once a hair cell has been transformed into an iPS cell, it can be coaxed to differentiate into virtually any cell in the human body.

A young woman is back home after a desperate journey to China for experimental stem cell treatment to heal her brain.

It wasn’t a disease or a genetic defect that led to her condition. Instead, Jackie Murphree, 22, went into a coma and woke up unresponsive and in need of wheelchair after a drunk driver struck her car.
The Decatur High honor student turned Aggie was leaving for work one morning two years ago when the accident occurred. The driver got probation.

In the latest on her slow and painful journey to find the person she used to be, Jackie went to China for five weeks. Doctors injected her own stem cells into her spinal cord in an attempt to heal her brain. She returned to North Texas Wednesday.
Patrick Murphree, her father, has been in the forefront of the battle, leading the quest to find help for his daughter. In therapy each day, he said he hopes each small response is a sign that his daughter is stepping closer to who she was before the crash.

“When she’s having a bad day, and she’s obviously in pain, I get real mad that she’s having to go through this,” he said.
Her physical therapist, Merley Vela, said she has seen through the way Jackie holds her head up or leans on her arm that there are improvements.

“Before she could not hold her head up,” Vela said. “Before she did not react at all. But now she gets angry. You can see her facial expression when she’s mad.”
Murphree quit his job to care for his daughter while Jackie’s mom supports the family.

“This is my job,” he said. “This is what I do. I don’t do anything else. It’s one of those things where we don’t know what’s going to happen tomorrow, but it doesn’t change what I do today.”
But her parents haven’t been the only one supporting Jackie’s journey. Hundreds of neighbors in Decatur raised more than $30,000 for experimental treatment in China that is not available in the Unites States.

“We don’t know if she’s on one of her peaks, or it’s the result of what happened,” Murphree said of watching his daughter over the last week react more to conversation.
Not expecting an overnight miracle, Jackie’s family has been cherishing the small signs.

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