A recent study by Scripps scientists offers good news for families of children afflicted with the rare genetic disorder, cystinosis. In research that holds out hope for one day developing a potential therapy to treat the fatal disorder, the study shows that the genetic defect in mice can be corrected with stem cell transplantation.... A research team used bone marrow stem cell transplantation to address symptoms of cystinosis in a mouse model. The procedure virtually halted the cystine accumulation responsible for the disease and the cascade of cell death that follows.
http://www.sciencedaily.com/releases/2009/09/090917131656.htm
http://www.sciencedaily.com/releases/2009/09/090917131656.htm