New Gene Editing Strategies used to Treat Rare Genetic Diseases

 A recent article by the New York Times, entitled “New Gene-Editing Strategy Could Help Development of Treatments for Rare Diseases”, discusses a new approach to genetic editing. This new technology is led by Dr. David Liu, a biologist at the Broad Institute and Harvard. Currently, the process of gene editing offers hope for treating certain genetic diseases; however, it is both demanding of resources and time and only applies to a small number of patients. With new research methods, Dr. Liu is hopeful for a more efficient method of gene editing. Instead of using personalized gene editing for each disorder, Dr. Liu's research provides a pathway for a more standard way to correct genetic diseases. This technique could treat patients regardless of what genetic mutation they suffer from. 7,000 total rare genetic diseases affect more than 400 million individuals worldwide. This study is focused on “nonsense mutations” that cause the production of premature stop codons, causing the protein to be truncated. Dr. Liu's technology inserts a molecule that replaces the stop codon and continues manufacturing a full-length protein, instead of targeting the specific mutation itself. The future for this technology is long and must be well tested before it can be seen in human patients. However, there is great promise for the future of gene editing and future patients. Before this technology, gene editing was used to alter specific mutations in individual patients. A case of gene editing in a baby at the Children's Hospital of Philadelphia shows the success of this genetic editing on a baby born with a rare genetic mutation that usually kills 50% of infants born with this mutation. An article describing this case shows this infant as one of the first to be cured successfully by genetic engineering. This success provides hope for the future of genetic editing and the potential future outcomes that may stem from this success. 

This new technology enhances the process of genetic editing. This gives great hope for the future of science and medicine as genetic editing for specific mutations has already been proven successful, highlighted in the case of the infant whose life was saved from such a process. Hopefully, in the future, this efficient and cost-effective technology can serve the 400 million people suffering from rare genetic diseases around the world.