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.
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.
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.
A fourteen-year-old cancer patient has gone into full remission after partaking in the clinical trial of a stem cell therapy conducted at the Children’s Hospital of Philadelphia. The recently approved FDA treatment, also known as chimeric antigen receptor T-cell [CAR T cell] therapy, works by obtaining autologous (the patient’s own) immune stem cells, genetically altering and expanding them to recognize a specific molecule on the surface of cancer cells and become targeted cancer killers. In this case, it was used to treat acute lymphocytic leukemia (ALL) originating in the B cells.
Researchers at UCLA have developed a ‘bionic thymus’ capable of transforming blood stem cells into T cells of the immune system that can be targeted to attack cancer cells. During the transformation, the researchers were also able to incorporate a tumor-targeting gene in anticipation of utilizing the cells to fight cancers. T Cell production is a long and complex biological process in the body and many cancer patients may not have enough of their own T cells to collect and direct to combat their cancer. Therefore, the creation of an artificial thymus has the potential to resolve this issue. The process also shuts off the expression of normal T cell surface receptors, which the researchers believe may enable the cells to be used by other patients without the risk of rejection.