Two Department of Pediatrics Researchers Earn “Perfect 10s” from the NIH
Congratulations to Barry Byrne, M.D., Ph.D., and Roland Herzog, Ph.D. on this rare achievement.
Overview of “Perfect 10”
The scoring system, which is peer reviewed, utilizes a nine-point rating scale with one equivalent to exceptional and nine being poor. The final overall score is determined by multiplying the average score by 10. The final overall scores range from 10, or high impact, through 90, or low impact. Thus, earning a “Perfect 10” equates to a perfectly scored project and landing in the top one percentile of applications.
The scoring criteria include review of the project’s significance, investigators, innovation, approach and environment. The two components with the heaviest weighting are significance and approach, which address how scientific knowledge, technical capability and clinical practice will be improved if the aims are achieved, as well as the overall strategy, methodology and analyses of the project.
Barry Byrne, M.D., Ph.D.
Byrne’s, project is centered on Pompe disease, which is a lysosomal storage disorder associated with systemic deficiency of an enzyme that is required to degrade glycogen. The continuing research project, which is a joint program with David Fuller, Ph.D., in the Department of Physical Therapy and Mai ElMallah, M.D., a Department of Pediatrics Pulmonary Fellow, hypothesizes that part of the reason that breathing problems are common in Pompe disease is that brainstem neurons and spinal cord motoneurons fail to adequately control the respiratory muscles. Byrne’s research targets optimization of adeno-associated virus, or AAV, based therapies to treat respiratory neurons in Pompe disease.
Visit the Powell Gene Therapy Center website for additional information on Byrne’s research.
Roland Herzog, Ph.D.
Dr. Herzog’s project aims to develop novel gene therapy treatments to prevent or eliminate undesired immune responses in the treatment of hemophilia, an inherited bleeding disorder. Immune responses to coagulation factors can cause complications in the treatment of patients with hemophilia, which lead to increased morbidity and mortality, as well as a substantially increased treatment costs. The ideal treatment would involve an injection of a harmless vector delivering a therapeutic gene to enable the body’s cells to produce the missing clotting factor indefinitely. Such a gene therapy that also promotes immune tolerance would substantially improve the lives of hemophilic patients and lower health care costs.
Visit the Herzog Lab website for additional information on his research.
What is the broader significance of earning a “Perfect 10?” Well, apart from being highly esteemed, the better the score, the higher the likelihood that the project will get funded. So, our “Perfect 10” researchers are very pleased with the results, to say the least.