Gene Therapy in Pediatric Orthopedics

Abstract

Gene therapy is gaining traction as an effective treatment for several deleterious disorders by delivering genetic material using viral or non-viral vectors to correct mutated genes. Research in the field focuses primarily on the treatment of cancers; however, it shows great promise for treating diseases related to pediatric orthopedics. This review aims to describe gene therapy’s application, efficacy and safety in pediatric orthopedics. This paper will examine common pediatric orthopedic disorders including Duchenne muscular dystrophy, osteogenesis imperfecta, spinal muscular atrophy and osteosarcoma. Overall, gene therapy for spinal muscular atrophy and Duchenne muscular dystrophy has made great advances with approved gene therapy drugs already in use, while therapy for osteogenesis imperfecta and osteosarcoma treatments is still widely preclinical but still promising. As a whole, gene therapy is rapidly advancing in the field of pediatric orthopedics; however, further research is crucial in continuing and spreading these advancements and for the treatment of other debilitating pediatric-related orthopedic disorders.

Keywords

• gene therapy
• pediatric orthopedics
• osteogenesis imperfecta
• Duchenne muscular dystrophy
• osteosarcoma
• spinal muscular atrophy