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 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 recent article in the NY Times served to highlight the progress the medical community is making in combating genetic disorders.
The development of Crispr-Cas9 gene editing technology is profoundly altering the way the medical community is approaching the treatment of genetic disorders. By enhancing accuracy and simplifying the process for the removal and insertion of specific genes in the DNA sequence, Crispr-Cas9 has brought gene therapy back to the forefront of research in the treatment of genetic disorders.To no surprise, one of the key components of this treatment approach are stem cells.
A recent clinical trial conducted at Great Ormond Street Hospital in London, England, has successfully treated seven patients suffering from the genetic disorder Wiskott-Aldrich Syndrome by utilizing autologous [the patient’s own] stem cells. The therapy was an example of translational genomics, in which doctors extract autologous stem cells, correct the faulty gene that causes Wiskott-Aldrich Syndrome, and then implant the stem cells back into the patients to produce new, healthy cells.