Salk researchers unveil breakthrough on genetic diseases

Researchers at the Salk Institute for Biological Studies in La Jolla, CA, have combined stem cell and gene therapy techniques into a new approach that could point the way to a cure for some of the world's most devastating ailments.

Their research project involved taking a defective cell from patients suffering from Fanconi anemia--a genetic disease that can trigger leukemia and other fatal diseases--and correcting the defective gene that causes the condition. The repaired cells were then made into induced pluripotent stem cells, or iPS cells, which readily differentiated into hematopoietic progenitor cells primed to differentiate into healthy blood cells.

"We haven't cured a human being, but we have cured a cell," says the study's leader, Juan-Carlos Izpisúa Belmonte. "In theory we could transplant it into a human and cure the disease."

"If we can demonstrate that a combined iPS-gene therapy approach works in humans, then there is no limit to what we can do," says Inder Verma, who worked on the project.

- check out the press release

Suggested Articles

Compass' CD137 agonist cleared large tumors in mice that other I-O agents had failed to treat. It's advancing the drug into phase 1 human trials.

UPMC researchers are planning clinical trials of a COVID-19 vaccine that uses pieces of the virus' spike protein to create immunity.

Treating mice with niacin increased the number of immune cells in glioblastomas, reducing tumor size and extending survival.