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Osteogenesis Imperfecta (OI) describes a series of genetic bone fragility disorders that can substantially impact patient quality of life. The multidisciplinary approach to managing children and adults with OI primary involves the administration of anti‐resorptive medication, allied health (physiotherapy and occupational therapy), and orthopaedic surgery. However, advances in gene-editing technology and gene therapy vectors promise gene‐targeted interventions to provide an enduring or perhaps permanent cure for OI. This review describes emergent technologies for cell and gene‐targeted therapies, significant hurdles to their implementation, and discusses the prospects of their future success focusing on bone disorders.
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