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Review
. 2022 May;37(5):826-836.
doi: 10.1002/jbmr.4549. Epub 2022 Apr 17.

Curative Cell and Gene Therapy for Osteogenesis Imperfecta

Aaron Schindeler  1   2 , Lucinda R Lee  1   2 , Alexandra K O'Donohue  1   2 , Samantha L Ginn  3 , Craig F Munns  4   5   6
Affiliations
Review

Curative Cell and Gene Therapy for Osteogenesis Imperfecta

Aaron Schindeler et al. J Bone Miner Res. 2022 May.

Abstract

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).

Keywords: CELL THERAPY; COLLAGEN; GENE THERAPY; OSTEOGENESIS IMPERFECTA.

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Conflict of interest statement

The authors have received philanthropic foundation from the Teicke Foundation, the Flicker of Hope Foundation, the Sydney Children's Hospitals Foundation, and have been previously supported by the Care 4 Brittle Bones Foundation, Sticks and Stones Foundation, and Children's Tumor Foundation.

Figures

Fig. 1
Fig. 1
Osteogenesis imperfecta–associated genes, their cellular location and mechanism of action within osteoblasts.
See this image and copyright information in PMC

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