Hematology

Sickle cell disease corrected in human models using stem cell-based gene therapy

Published in Gene Therapy Weekly, January 26th, 2006

In a study to be published in the Nature Biotechnology, researchers led by a team of scientists at Memorial Sloan-Kettering Cancer Center have devised a strategy that uses stem cell-based gene therapy and RNA interference to genetically reverse sickle cell disease (SCD) in human cells.

This research is the first to demonstrate a way to genetically correct this debilitating blood disease using RNA interference technology.

To prevent the production of the abnormal hemoglobin that causes sickle cell disease, a viral vector was introduced in cell cultures of patients who have the disease. The vector carried a therapeutic globin gene harboring an...

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Source: Gene Therapy Weekly (2006-01-26)

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