Researchers at USC [University of Southern California] have utilized stem cells to track neuronal growth and identify specific genes that appear to be responsible for the development of schizophrenia, bipolar disorder and depression. The study linked the DISC1 gene to the development of schizophrenia, which currently does not have effective treatments and causes disproportionate disability compared to other neurological disorders. Like many neurological disorders, the source of schizophrenia has been ambiguous and this research, with the use of stem cells, is helping to navigate this disorder. Through the utilization of stem cells, the study determined how genes like DISC1 function in the body, and their downstream impact on protein function and neurotransmitter production by tracking the gene expression.
Researchers at Newcastle University are 3D printing corneas utilizing stem cells. The process involves mixing stem cells in a bio-gel which is derived from seaweed and collagen that allows these stem cells to be cultured and printed easily and efficiently into fully functioning corneas. The cornea plays an important role in focusing light that enters the eye. Technically, blindness caused by corneal damage is easily reversible with a corneal transplant. However, there is a vast shortage of donor corneas due to general organ and tissue donation shortages. In addition, there is also a significant risk of rejection - as is the case with any donated tissue.
“Clean meat” company Future Meat Technologies anticipate they can bring the price of lab-grown, “meatless” meat down to approximately $8 per kg [$4 per pound]. The process involves obtaining mesenchymal stem cells from the animal and differentiating the stem cells into both muscle and fat tissues, which are indistinguishable from those found in standard meat. The meat cooks, tastes and smells exactly like anything you’d get from an animal- however, the biggest hurdle has been its high price. Future Meat Technologies looks to overcome this hurdle by bringing costs down, by differentiating stem cells more efficiently and scaling up production.
A mesenchymal stem cell treatment for patients with cardiac muscle degeneration and ventricular failure is being conducted at the MedStar Heart and Vascular Institute. The patients currently being recruited for the study are those who require left ventricular assist devices (LVADs) in order to pump their heart, and these patients are in severe stages of cardiac failure. In pre-clinical models, intravenous mesenchymal stem cell injections have greatly improved left ventricular function, which is responsible for pumping and pressurizing the blood to the rest of the body. Additionally, there was a significant decrease in the inflammatory response that is indicative of damaged cardiac muscles. By reducing inflammation researchers hope to not only provide immediate relief for the strained cardiac muscle, but also slow or stop the progression of heart failure.
A collaborative effort between researchers at Stanford University, the Joint Institute of Metrology and Biology, and the National Institute of Standards and Technology has developed a modified and more targeted version of CRISPR, which is more efficient at editing single nucleotide mutations. The new system is called MAGESTIC (multiplexed, accurate genome-editing through short, trackable, integrated cellular barcodes), and it has been shown to successfully modify genes by accurately targeting the location of defective genes. MAGESTIC ameliorates and addresses the current shortcomings of gene-editing technology by enhancing the ability of CRISPR to target single genes [out of millions] with the purpose of correcting specific mutations.
A genetically modified stem cell therapy for Diffuse Large B-Cell Lymphoma (DLBCL) has been approved by the FDA. Researchers at the Abramson Cancer Center, in collaboration with Novartis, have successfully administered a CAR-T Cell therapy, called Kymriah, for the most common type of non-Hodgkin Lymphoma. DLBCL is a fast growing cancer that affects B lymphocytes, which are responsible for producing antibodies that help fight infections in the body. This groundbreaking treatment involves obtaining autologous (the patient’s own) T cells, which are a more specialized type of stem cell, and genetically engineering the cells to track down and destroy cancerous cells.
Researchers at the Salk Institute are developing an autologous stem cell cure to treat hemophilia, a genetic disorder affecting millions worldwide. Hemophilia is a disorder in which a person’s blood has a diminished ability to clot, posing the risk of severe bleeding from minor injuries like nosebleeds. Additionally, people with hemophilia are at an even greater risk for internal bleeding, which can arise from minor injuries. Hemophilia is typically inherited but can also be acquired in adulthood. The genetic disorder is caused by an inappropriate immune response where immune cells attack the blood’s clotting factors, or a mutation that prevents the production of the clotting factor altogether. This treatment involves obtaining autologous (the patient’s own) stem cells, editing them to correct the faulty gene with the help of CRISPR (a gene editing technology), and reintroducing the cells back into the body.
Legendary golfer Jack Nicklaus is back on the course, thanks to stem cells. After years of debilitating chronic back pain that limited his playing time, the autologous [his own stem cells] treatment has him back swinging and playing competitively again. By utilizing his own stem cells, Jack virtually eliminated any chance of rejection, thereby significantly increasing the odds of a successful outcome.
A Phase I clinical trial to test the efficacy of genetically modified autologous (the patient’s own) stem cells to treat beta-thalassemia has been initiated. This condition is an inherited disease that affects the production of hemoglobin, which is responsible for carrying oxygen in the body and delivering it to tissues and vital organs. With thousands of new cases every year, this condition often results in fatigue, bone fragility and extreme anemia (a deficiency of iron in the blood). This trial aims to create a groundbreaking protocol that would obtain autologous stem cells from the patients, genetically alter them to produce the missing protein responsible for the condition and, reintroduce the stem cells back into the body through a transfusion.