Curative Cell and Gene Therapy for Osteogenesis Imperfecta
Summary
Osteogenesis imperfecta (OI) describes a series of genetic bone fragility disorders that can have a substantive impact on patient quality of life. The multidisciplinary approach to management of children and adults with OI primarily involves the administration of antiresorptive medication, allied health (physiotherapy and occupational therapy), and orthopedic surgery. However, advances in gene editing technology and gene therapy vectors bring with them the promise of gene-targeted interventions to provide an enduring or perhaps permanent cure for OI. This review describes emergent technologies for cell- and gene-targeted therapies, major hurdles to their implementation, and the prospects of their future success with a focus on bone disorders. © 2022 The Authors. Journal of Bone and Mineral Research published by Wiley Periodicals LLC on behalf of American Society for Bone and Mineral Research (ASBMR). © 2022 The Authors. Journal of Bone and Mineral Research published by Wiley Periodicals LLC on behalf of American Society for Bone and Mineral Research (ASBMR).
| Authors | Schindeler A, Lee LR, O'Donohue AK, Ginn SL, Munns CF |
|---|---|
| Journal | Journal of bone and mineral research : the official journal of the American Society for Bone and Mineral Research |
| Publication Date | 2022 May;37(5):826-836 |
| PubMed | 35306687 |
| PubMed Central | PMC9324990 |
| DOI | 10.1002/jbmr.4549 |