gamma-globin

Novel approaches to increase gamma-globin expression in sickle cell disease

Sickle cell disease research at The Children's Hospital 2010

Adult beta-globin gene expression in the genetically engineered mice went down 50 to 80 percent and fetal gamma-globin gene expression went up after removing ERV-9.

PhysOrg.com - latest science and technology news stories 2010

The researchers also transplanted the altered blood-forming cells from the original treated mice into a second generation of sickle cell mice to show that the gamma-globin gene had incorporated itself permanently into the blood-forming cells.

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The investigators found that months after they introduced the altered blood-forming cells, the mice continued to produce gamma-globin in their red blood cells.

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"The successful introduction of the gamma-globin gene into the stem cells of the sickle cell disease mouse practically cured them," said Tamara Pestina, PhD, Hematology, the paper's first author.

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The scientists introduced the gene for gamma-globin into the mice's blood-forming cells and then introduced those altered cells into the mice.

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This immature hemoglobin, which usually disappears after birth, does not contain beta-globin, but another form called gamma-globin.

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"The successful introduction of the gamma-globin gene into the stem cells of the sickle cell disease mouse practically cured them," said Tamara Pestina, PhD, Hematology, the paper's first author.

unknown title 2009

He and his colleagues developed a technique to insert the gene for gamma-globin into blood-forming cells using a harmless viral carrier.

unknown title 2009

The researchers also transplanted the altered blood-forming cells from the original treated mice into a second generation of sickle cell mice to show that the gamma-globin gene had incorporated itself permanently into the blood-forming cells.

unknown title 2009