Curative Cell and Gene Therapy for Osteogenesis Imperfecta.
Aaron SchindelerLucinda R LeeAlexandra K O'DonohueSamantha L GinnCraig Frank MunnsPublished in: Journal of bone and mineral research : the official journal of the American Society for Bone and Mineral Research (2022)
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).
Keyphrases
- gene therapy
- bone mineral density
- bone regeneration
- healthcare
- genome wide
- bone loss
- soft tissue
- single cell
- copy number
- public health
- minimally invasive
- primary care
- postmenopausal women
- current status
- cell therapy
- emergency department
- physical activity
- stem cells
- dna methylation
- young adults
- machine learning
- gene expression
- climate change
- case report
- transcription factor
- health information
- coronary artery disease
- big data
- rectal cancer
- prognostic factors
- genome wide analysis
- drug induced