Science Rocks features AbbVie scientists who share interesting research in their field and why it matters. In this month's feature, Christopher Miller, Ph.D, a director in The Genomics Research Center within target enabling science and technologies, recently discussed gene editing at the Cleveland Clinic Medical Innovation Summit. After his presentation, he stuck around to hear the top 10 medical innovations for 2018, as predicted by the Cleveland Clinic. Here’s what he thinks:
The world’s first artificial pancreas.
A wearable patch to help sleep apnea.
Gene therapy for blindness.
The Cleveland Clinic unveiled their top 10 medical innovations of 2018 at this year’s Medical Innovation Summit. At the Summit, I sat on a panel that discussed gene editing and gene therapy, but was also interested in their list of potential breakthroughs for next year.
Being a scientist, of course, I have some thoughts of my own. While this year’s innovations cover a variety of potential innovations including pharmaceutical therapies, medical devices and medical practices and procedures, one thing is clear – all have the potential to profoundly impact patient care and treatment.
Each of the new pharmaceutical therapies could be game-changing. The field of gene therapy has made important advances, but has also suffered some high profile setbacks. Gene therapy to treat eye diseases has some advantages based on accessibility and anatomy of the eye and the existence of several genetically-based eye diseases for which replacement of a defective gene has been shown to be curative. The best known examples of this are Leber Congenital Amaurosis and Retinitis Pigmentosis, both caused by RPE65 mutations. Gene therapy to deliver normal RPE65 has been shown to overcome disease in human clinical trials with FDA approval anticipated in 2018. If this happens, it would be a win for the gene therapy field and for patients suffering from these diseases.