Duchenne Muscular Dystrophy, a particularly severe form of muscular dystrophy [MD – a genetic disorder], affects as many as 15,000 young Americans. Many of those afflicted eventually succumb to cardiac or respiratory failure by their early 20’s. Researchers at University of California, Los Angeles, are utilizing stem cells to model the disease in order to develop and test treatments. One particularly promising treatment involves a method referred to as exon skipping. The technique essentially tricks the cell’s machinery into misreading the MD genetic mutation so instead of producing the defective protein responsible for the disease, the cells produce a more functional version of the protein. In using muscle cells derived from stem cells of people with muscular dystrophy – the cells contain the mutation that causes muscular dystrophy – researchers anticipate their tests to more accurately reflect how human cells would react to their drugs [or combination of drugs]. This enables more efficient and effective testing of potential treatments and speeds the process of developing those treatment options that show the most promise. According to Stanley Nelson, a lead researcher on the project, “We are thrilled that stem cell research will change the outcome of Duchenne”.
Scientists at the University of California Davis’ Institute for Regenerative Cures are utilizing mesenchymal stem cells [the same type of stem cells found in teeth] to develop a new therapy that targets the genetic abnormality in Huntington’s disease. The principal investigator of the study, and the director of UC Davis stem cell program and the UC Davis Institute for Regenerative Cures, Jan Nolta said, “Our team has made a breakthrough that gives families affected by this disease hope that genetic therapy may one day become a reality.” The treatment seeks to address the root cause of the disease as opposed to merely mitigating the symptoms of the disease.
Scientists in the US (UC, UCSD) and China (Wuhan) have found a way to convert stem cells into functional neurons. The researchers were able to suppress an RNA-binding protein, inducing the stem cells to become neurons. This gives hope for a treatment for neurodegenerative diseases such as Huntington’s, Parkinson’s, Alzheimer’s, Multiple Sclerosis, and Amyotrophic Lateral Sclerosis (ALS or Lou Gehrig’s disease), which will afflict one in four Americans over their lifetime.
Researchers at the University of California, San Diego and the University of Sheffield have worked together to find a way to improve stem cell scaffolding through sticky spots scattered throughout the extra-cellular matrix. The improvement in scaffolding will lead to better stem cell treatments in many areas. Right now, scaffolds are being used in tandem with stem cells to grow new tracheas, teeth, cartilage, organs and much more.