Researchers at ETH Zurich have developed advanced CRISPR gene editing technology to modify an entire gene network in one shot. This is a significant step forward in correcting genetic disorders, as many genetic abnormalities and mutations that lead to palpable symptoms are controlled by several genes in various locations on the genome. The advance resulted from the use of a CRISPR enzyme called Cas12a, as opposed to Cas9, which is currently used for all gene editing done with the CRISP technology.
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 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.