Tag Archives: Genetic disorder

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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Stem cells to heal blood disorders

Scientists and transplant clinicians at the Ansary Stem Cell Institute at Weill Cornell Medical College and the Center for Cell Engineering at Memorial Sloan Kettering Cancer Center have been awarded a $15.7 million, four-year research grant from the New York State Stem Cell Science Program (NYSTEM).

The scientists will translate their approach to manipulate hematopoietic stem cells to cure acquired and inherited blood disorders. For many patients with such blood diseases, including sickle cell disease, the only hope for a cure requires transplanting normal blood stem cells.

But in many instances suitable blood stem cells cannot be found or there are
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