Stem Cell Research: Doctors at the Sutter Neuroscience Institute in Sacramento, California have started a new FDA-approved clinical trial examining the utility of cord blood in the treatment of autism. Cord blood contains stem cells, a unique cell type that can turn into many different kinds of cells in the body and communicate with other cells in ways we do not fully understand. This section of Autism Reading Room will help you understand how stem cell research is providing new and exciting information about autism diagnosis and treatment.

We all begin as stem cells. Very early during development, a small population of cells, called embryonic stem cells, hold the potential to become almost every type of cell in the body. In 2006, a group of researchers in Japan reported a way to convert cells from the skin back into stem cells. The conversion process enables neurons to be made from stem cells derived from the skin of a person with autism. This groundbreaking study removed a difficult obstacle to ASD research—studying the neurons of a person with autism—by enabling neurons to be grown from stem cells in a petri dish. Scientists are now using stem cells to investigate the inner workings of neurons from people with autism to develop new, individualized diagnostic and therapeutic tools.

Key Points

• Stem cells are capable of becoming many cell types in the body, including neurons and other types of cells in the brain.
• The ability to generate induced pluripotent stem cells (iPSCs) from the fibroblasts of human patients and subsequently differentiate them into particular cell types has allowed researchers a means to bridge the gap between cellular phenotype and clinical phenotype.
• iPSC technology have been used to generate and examine cellular defects in neurons from patients with monogenic syndromes associated with ASD, such as Rett syndrome and Timothy syndrome.
• Stem cell research can be used in drug screening of iPSC-derived brain cells from ASD patients and in the potential development of individualized therapies.

Misconception

Embryonic stem cells are taken from eggs that have been fertilized within a woman’s body.

Fact

Embryonic stem cells are derived from donated preimplantation embryos that have been fertilized outside of the body in fertilization clinics.