The first steps on the gene therapy pathway to anti-sickling success

Article Abstract:

Researchers have developed a mouse model of sickle cell disease that can be used to estimate how much fetal hemoglobin is needed to prevent hemoglobin polymerization. Once this is known, gene therapy could be used to give patients the gene for fetal hemoglobin.

Models

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Genetic correction of sickle cell disease: insights using transgenic mouse models

Article Abstract:

Giving sickle cell patients the gene for fetal hemoglobin could prevent hemoglobin polymerization. In a mouse model, a fetal hemoglobin concentration between 9% and 16% of total hemoglobin improved all hematologic parameters as well as life span.

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Gene therapy of experimental brain tumors using neural progenitor cells

Article Abstract:

Researchers treated glioblastomas in mice using neural stem/progenitor cells containing the gene for interleukin-4. The progenitor cells can travel to areas of the brain that are difficult to reach using viral vectors.

Stem cells, Glioblastoma multiforme, Interleukin-4

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