Stem Cell and Gene Therapy for Sickle Cell and other Genetic Diseases
By Jeffrey Norris | December 13, 2011
A cure for sickle cell anemia and other life-threatening genetic disorders that arise in the blood is the goal of a new $6.7-million, five-year research project headed by UCSF scientist Y. W. Kan, a pioneer of modern genetics and the diagnosis of genetic diseases before birth.
The pre-clinical research is funded through a competitively awarded grant from the National Institutes of Health (NIH).
The new approach to be explored by Kan and UCSF collaborators, including Dieter Gruenert, PhD, and Marcus Muench, PhD, is based on stem cells and gene therapy. The UCSF researchers will primarily work with stem cells derived by manipulating and reprogramming cells from adults — not with embryonic stem cells. In clinical practice these stem cells would be made from a patient’s own tissue, and the inborn genetic error then would be corrected through genetic engineering.
The procedure that the UCSF scientists are developing is expected to offer a way to circumvent the need to find immunologically compatible bone marrow donors, and eliminate the threat of graft versus host disease — because use of stem cells derived from the patient should not cause unwanted immune responses when put back into the patient.
“This project offers a possibility of curing both newborn and adult patients with their own cells that have been reprogrammed, corrected and converted to cells that will regenerate all of their blood cells, including the immune system,” Kan said.
Kan and colleagues aim to convert a patient’s blood cells into powerful “pluripotent” stem cells. The researchers then will replace a defective portion of a gene in the stem cells using experimental techniques. Finally, they will coax the cells to become the kind of more specialized stem cells — called hematopoietic stem cells — that can specifically regenerate the entire range of blood cells.