A team of researchers at the UC Davis Institute for Regenerative Cures has developed a technique for using stem cells to deliver therapy that specifically targets the genetic abnormality found in Huntington’s disease, a hereditary brain disorder that causes progressive uncontrolled movements, dementia and death. The findings, now available online in the journal Molecular and Cellular Neuroscience, suggest a promising approach that might block the disease from advancing.
“For the first time, we have been able to successfully deliver inhibitory RNA sequences from stem cells directly into neurons, significantly decreasing the synthesis of the abnormal huntingtin protein,” said Jan A.
In an effort to identify the underlying causes of neurological disorders that impair motor functions such as walking and breathing, UCLA researchers have developed a novel system to measure communication between stem cell–derived motor neurons and muscle cells in a Petri dish.
The study provides an important proof of principle that functional motor circuits can be created outside the body using these neurons and cells and that the level of communication, or synaptic activity, between them can be accurately measured by stimulating the motor neurons with an electrode and then tracking the transfer of electrical activity into the muscle cells
Stem-cell science is one of the most promising areas of biomedical research with the potential to dramatically improve treatment of the most intractable diseases, but it will likely take several more years to fully realize its potential.
That was the message at the annual Ansary Symposium on Stem Cell Research on June 6. The day-long event, which marked the 10th anniversary of the creation of the Ansary Stem Cell Institute at Weill Cornell Medical College, assembled the country’s leading stem-cell investigators to discuss the field’s achievements, its challenges and future.
“The potential for stem cells is tremendous: If someone has a