Mayra Pellati/USP
Human stem cells used to treat muscular dystrophy in golden retrieversMayra Pellati/USPAn experiment performed on five dogs indicates that it may be safe to use human stem cell transplantation to treat Duchenne muscular dystrophy, the most common and most severe form of muscular degeneration (Stem Cell Reviews and Reports, August 2016). A team from the Institute of Biomedical Sciences at USP (ICB/USP), coordinated by geneticist Mayana Zatz, derived adipose stem cells from human donors, grew them, and then transplanted them to five golden retrievers with muscular dystrophy and intact immune systems. This breed is sometimes born with a genetic mutation that causes a form of dystrophy similar to the human disease, making long-term follow-up possible. Over six months, nine applications of the cells were injected into a vein in the dog’s front paw. The dogs received their first shot at the age of 2 to 3 months. Clinical exams, blood tests, imaging, and cardiac assessments indicated that the treatment was well tolerated and apparently had no unwanted long-term effect; follow-up lasted 7 years. The researchers attribute the longevity of two of the dogs to the benefits of the treatment. Animal research is important both for treating dogs and for verifying therapeutic safety prior to human clinical trials. Duchenne muscular dystrophy is hereditary and can cause loss of movement as early as the age of 10.
Republish