There are endless genetic diseases, such as cystic fibrosis, muscular dystrophy or even spinal muscular atrophy, where a cure is yet to be found. However, Gang Bao and his team of bioengineers at Rice University have developed a potential new gene therapy for these incurable genetic disease by utilizing moths and magnets. This new technology combines a virus that infects the North American moth, Autographa californica, and nanoparticles to potentially repair the mutations that cause genetic diseases. The moth infecting virus is crucial in this experimental research for it serves as a carrier for CRISPR/Cas9, which is an system consisting of an enzyme and piece of RNA that is often used as a genome editing tool. The magnetic field works to manipulate gene expression of the virus, which is normally inactive in blood, in the targeted tissues. Nevertheless, the main challenge to their work was finding a process in which CRISPR/Cas9 can be delivered to the target area with high efficiency. When working to solve this roadblock, the team had to ensure that only the target area was affected because altering the DNA of other tissues or organs could be detrimental.
Bao’s group developed a way around this problem by specifically choosing the moth-infecting virus. This virus contains cylindrical baculovirus vector (BV), which is the part of the virus that acts as a unit to carry the CRISPR/Cas9. The BV is able to transport the CRISPR/Cas9 to the target site since there naturally exists a protein (C3) which inactivates the BV. Once the BV reaches the site of interest, a magnetic field is applied and is then able to overcome the deactivation caused by C3. Thus, the CRISPR/Cas9 can then serve its purpose of repairing the mutations that give rise to the genetic diseases.
I feel as if this work is important to the scientific community’s understanding of incurable diseases even if it does not end up being the be all end all answer to stopping the diseases entirely. This work will definitely contribute in enhancing gene therapies and at least reduce the side effects of the disease while making life on those facing these challenges easier. Personally, the most interesting aspect of article was that during the testing process the team had used Green Fluorescent Proteins (GFP) to determine if the use of magnets contributed to increasing delivery efficiency of BV to the target site. Since students have been working with GFP in the laboratory, it was a nice to see how work done within the weekly labs can be related to groundbreaking work done out in the field.