Curative cell and gene therapy for osteogenesis imperfecta

Schindeler, Aaron; Lee, Lucinda R.; O'Donohue, Alexandra K.; Ginn, Samantha L.; Munns, Craig F.

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

Curative cell and gene therapy for osteogenesis imperfecta

J Bone Miner Res. May 2022;37(5):826-836

Affiliations

¹Bioengineering and Molecular Medicine Laboratory, the Children’s Hospital at Westmead and the Westmead Institute for Medical Research, Westmead, Australia

²Children’s Hospital Westmead Clinical School, University of Sydney, Camperdown, Australia

³Gene Therapy Research Unit, Children’s Medical Research Institute, Faculty of Medicine and Health, The University of Sydney and Sydney Children’s Hospitals Network, Westmead, Australia

⁴Faculty of Medicine, The University of Queensland, Brisbane, QLD, Australia

⁵Department of Endocrinology and Diabetes, Queensland Children’s Hospital, Brisbane, QLD, Australia

⁶Child Health Research Centre and Faculty of Medicine, The University of Queensland, Brisbane, Queensland, Australia
Abstract and keywords

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.

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

DOI: 10.1002/jbmr.4549

PubMed: 35306687

WoS: 000783068100001
History

Received: 2021-11-04

Revised: 2022-02-03

Accepted: 2022-02-27

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