Vockley Published in Genetics in Medicine on the Increasing Role of Gene Therapy

Pitt Pediatrics congratulates Jerry Vockley, MD, PhD, FACMG, on his recent publication in Genetics in Medicine, which is an official journal of the American College of Medical Genetics. Titled “The evolving role of medical geneticists in the era of gene therapy: An urgency to prepare,” Vockley presents that the increasing role gene therapy will take in managing and treating rare diseases is rapidly turning the corner since its initial development in the late 1960s. 

While gene therapy has not yet reached an inflection point where it is routinely used to address genetic diseases, it can effectively be modeled on other single treatment care pathways, such as organ transplantation or stem cell transplantation. 

Despite this, Vockley argues that there may be other models that just as successfully balance cost and effectiveness that remain to be determined as gene therapies become more widespread, which only speaks to the need for more medical geneticists in the future of medical care for genetic diseases. 

In concluding, Vockley argues that the time is now to begin better educating all clinicians (especially medical geneticists) in the fundamentals of gene therapy, as well as increasing the scope of training and development of new medical geneticists. As gene therapy treatment becomes more commonplace, those suffering from rare diseases will need increased specialty care, while experts in the field will require increased leadership in clinical implementation. 

Follow Pitt Pediatrics on Twitter for future updates about the development of medical genetics. And check back on our Division of Genetic and Genomic Medicine for more information on ongoing clinical and scientific research.