Researchers at the University of Alabama at Birmingham (UAB) are developing a gene therapy that could serve as a permanent cure for sickle cell disease (SCD).
LentiGlobin uses a viral vector to deliver a gene that makes healthy hemoglobin into the stem cell. According to UAB researcher Julie Kanter, the initial patients treated with LentiGlobin are beginning to show signs of producing stable amounts of normal red blood cells containing healthy hemoglobin.
“This is like coding new instructions into the cell,” says Kanter. “The vector can deliver more than one copy of the instructions to each cell — usually between one and four copies — so the cell can make more HbA [normal hemoglobin] than HbS [Sickle hemoglobin].”
The UAB researchers are continuing to develop the treatment for improved efficacy, safety and cost-effectiveness.
“We need the therapy to be affordable so that people everywhere living with this disease have the option for gene therapy,” says Kanter. “Right now, most people with sickle cell disease live in sub-Saharan Africa and in India. They don’t have even the basic treatments they need like vaccines, penicillin or hydroxyurea that can make a huge difference in people’s lives with SCD. Eventually we need people in these areas to have equal opportunity to better outcomes.”
Source: Alabama Newscenter
