Potential implications of cell therapy for osteogenesis imperfecta

Research output: Contribution to journalReview article

18 Citations (Scopus)

Abstract

Osteogenesis imperfecta (OI) is a brittle-bone disease whose hallmark is bone fragility. Since the disease is genetic, there is currently no available cure. Several pharmacological agents have been tried with not much success, except the recent use of bisphosphonates. Stem cells have been suggested as an alternative OI treatment, but many hurdles remain before this technology can be applied for treating patients with OI. This review summarizes what is known at present regarding the application of stem cells to treat OI using animal models, clinical trials using mesenchymal stem cells to treat patients with OI and the knowledge gained from the clinical trials. Application of gene therapy in combination with stem cells is also discussed. The hurdles to be overcome to bring stem cells close to the clinic and future perspectives are discussed.

Original languageEnglish (US)
Pages (from-to)57-66
Number of pages10
JournalInternational Journal of Clinical Rheumatology
Volume4
Issue number1
DOIs
StatePublished - Jan 1 2009

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Osteogenesis Imperfecta
Cell- and Tissue-Based Therapy
Stem Cells
Clinical Trials
Inborn Genetic Diseases
Diphosphonates
Mesenchymal Stromal Cells
Genetic Therapy
Animal Models
Pharmacology
Technology
Bone and Bones

All Science Journal Classification (ASJC) codes

  • Rheumatology

Cite this

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title = "Potential implications of cell therapy for osteogenesis imperfecta",
abstract = "Osteogenesis imperfecta (OI) is a brittle-bone disease whose hallmark is bone fragility. Since the disease is genetic, there is currently no available cure. Several pharmacological agents have been tried with not much success, except the recent use of bisphosphonates. Stem cells have been suggested as an alternative OI treatment, but many hurdles remain before this technology can be applied for treating patients with OI. This review summarizes what is known at present regarding the application of stem cells to treat OI using animal models, clinical trials using mesenchymal stem cells to treat patients with OI and the knowledge gained from the clinical trials. Application of gene therapy in combination with stem cells is also discussed. The hurdles to be overcome to bring stem cells close to the clinic and future perspectives are discussed.",
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Potential implications of cell therapy for osteogenesis imperfecta. / Niyibizi, Christopher; Li, Feng.

In: International Journal of Clinical Rheumatology, Vol. 4, No. 1, 01.01.2009, p. 57-66.

Research output: Contribution to journalReview article

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AB - Osteogenesis imperfecta (OI) is a brittle-bone disease whose hallmark is bone fragility. Since the disease is genetic, there is currently no available cure. Several pharmacological agents have been tried with not much success, except the recent use of bisphosphonates. Stem cells have been suggested as an alternative OI treatment, but many hurdles remain before this technology can be applied for treating patients with OI. This review summarizes what is known at present regarding the application of stem cells to treat OI using animal models, clinical trials using mesenchymal stem cells to treat patients with OI and the knowledge gained from the clinical trials. Application of gene therapy in combination with stem cells is also discussed. The hurdles to be overcome to bring stem cells close to the clinic and future perspectives are discussed.

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