Osteogenesis imperfecta, rehabilitation medicine, fundamental research and mesenchymal stem cells.
A speculative suggestion for a curative approach to osteogenesis imperfecta is based on the experimental observation that a pluripotent stem cell for mesenchymatous tissues can be isolated from marrow or periosteum. This cell, referred to as a Mesenchymal Stem Cell (MSC) could provide a vehicle for gene therapy based on that fact that many mesenchymal tissues have reasonably high cellular turnover rates. If autologous MSCs with the "proper" gene construct can be transplanted into affected patients, it may be that the progeny of the gene-corrected MSC can replace the defective mature phenotypes and bring about curative events associated with normal cell turnover.
Caplan
A I
AI
Skeletal Research Center, Case Western Reserve University, Cleveland, Ohio 44106, USA.
D000818:Animals; D001861:Bone Regeneration / Q000502:physiology*; D019070:Cell Lineage / Q000502:physiology; D015316:Genetic Therapy / Q000379:methods / Q000639:trends; D006801:Humans; D045164:Mesenchymal Stem Cell Transplantation / Q000379:methods* / Q000639:trends*; D059630:Mesenchymal Stem Cells / Q000166:cytology / Q000378:metabolism*; D023421:Models, Animal; D010012:Osteogenesis / Q000502:physiology*; D010013:Osteogenesis Imperfecta / Q000628:therapy*