Punam Malik has led a team of researchers at the Cincinnati Children's Hospital Medical Center which has been studying the intricate workings of sickle cell anemia. And after a decade of work, he says that they're readying human trials of a new gene therapy that has the potential to cure the disease, which afflicts up to 100,000 African-Americans in the U.S.
People who suffer from sickle cell anemia carry a genetic defect that changes the shape of red blood cells, making it harder for them to do their work in transporting oxygen, leaving tissue damaged or dead. The research team has developed a new gene therapy that relies on autologous stem cells extracted from the bone marrow. The cells are implanted in the lab with an inactivated virus that carries a new gene that produces normal red blood cells. And that approach has been effective in curing the disease in animal models and tissue samples. Now the group is looking for regulatory approval to test their therapy in humans.
"If it works, it's a cure," said Malik, who runs the gene and molecular therapy research at Cincinnati Children's.
- read the article from the Cincinnati Enquirer