Breakthrough Gene Therapy Converts Harmful Fats, Targets Childhood Obesity and Arthritis

October 15, 2024
Breakthrough Gene Therapy Converts Harmful Fats, Targets Childhood Obesity and Arthritis
  • Currently, nearly 20% of children and teens in the U.S. are classified as obese, which significantly impacts their overall health.

  • Researchers at Shriners Children's St. Louis have developed a groundbreaking gene therapy that converts harmful omega-6 fatty acids into beneficial omega-3 fatty acids, potentially mitigating health risks associated with childhood obesity.

  • This innovative therapy employs an inactive adeno-associated virus (AAV) to deliver the gene responsible for this conversion, which could significantly impact children's health.

  • The research team, led by Dr. Farshid Guilak and Dr. Ruhang Tang, is optimistic that this gene therapy will not only reduce the incidence of arthritis in children but also enhance treatment effectiveness.

  • Their findings indicate that both obesity and the types of fatty acids prevalent in children's diets are major contributors to weight gain and associated health issues.

  • Dr. Guilak pointed out that the modern American diet is often high in omega-6 fatty acids, commonly found in fried foods and vegetable oils, while being low in omega-3s, which are beneficial for metabolic health.

  • Highlighting the obesity epidemic, Dr. Guilak emphasized that being overweight is the leading preventable risk factor for arthritis in children, underscoring the need for comprehensive solutions beyond mere weight loss.

  • The American Academy of Pediatrics supports early and intensive treatment for childhood obesity due to its links to serious health conditions, including arthritis and heart disease.

  • In preclinical studies, a single injection of the gene therapy was shown to improve metabolic health and reduce the effects of knee arthritis caused by a high-fat, omega-6-rich diet.

  • The study specifically addressed post-traumatic arthritis in children, often resulting from knee injuries, and demonstrated that the gene therapy could prevent diet-induced aging effects in mice.

  • Dr. Guilak's team is collaborating with the FDA to initiate clinical trials within the next three to five years, aiming to bring this promising therapy to children in need.

  • This research, which was funded by Shriners Children's and the National Institutes of Health, was published in the Proceedings of the National Academy of Sciences, marking a significant advancement in pediatric health.

Summary based on 3 sources


Get a daily email with more Science stories

More Stories