Tag Archives: Genetic disorder

Scientists create stem cells to help decipher diseases

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
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New Stem Cells Technique Offers Hope for Kids With Immune Deficiency

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

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.

Children with older father risk to develop malformations

Researchers now recognize that older age in a father can increase the risk that his children will develop a variety of disorders, including autism, schizophrenia, even a common form of dwarfism. The question is, how?

Now, in Stem Cell Reports, a research team has solved the problem for one such disease, Apert syndrome, and says its findings may extend to other paternal age-associated disorders. It is testing those disorders to see if that is true.

Scientists have for some time believed that the mutation for Apert syndrome — in which children are born with a disfigured skull, face, hands and feet
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Stem cell transplant can rectify infrequent genetic disorder in youths

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
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