Gene Therapy and Genome Editing in Rare Metabolic Bone Diseases: A Scoping Review of Current Progress and Future Perspectives

Objective: To map and synthesize the current evidence regarding gene therapy and genome editing applications for rare metabolic bone diseases, focusing on delivery vectors, editing technologies, and preclinical/clinical outcomes. Scope: This scoping review follows the JBI methodology for scoping reviews and the PRISMA-ScR (Preferred Reporting Items for Systematic reviews and Meta-Analyses extension for Scoping Reviews) guidelines. The review includes studies on Osteogenesis Imperfecta (OI), Hypophosphatasia (HPP), X-linked Hypophosphatemia (XLH), Fibrodysplasia Ossificans Progressiva (FOP), and Osteopetrosis. Methodology: Search Date: March 15, 2026. Databases: PubMed, Embase, and Scopus. Timeframe: 2010 – Present. Screening: A total of 772 records were identified after deduplication and will be screened for title and abstract, followed by full-text review using Covidence. Inclusion Criteria: Original research (preclinical in vivo/in vitro and clinical trials), focused on gene-based interventions, published in English.