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Abstract
Gene therapy defined as the transfer of gene or genetic material into a cell
with therapeutic intent. The genotype of this cell is altered, with subsequent
expression altering the phenotype of the cell. The therapeutic agent is gene
product, generally a protein or, less frequently, RNA. Skeletal gene therapy
can be applied independently or in conjunction with tissue engineering.
By delivering genes that promote healing directly to cells within the fracture site (in vivo gene therapy),or by genetically engineering bone marrow
stromal cells, muscle stem cells or other mesenchymal stem cell progenitors
to produce osteogenic factors prior to implantation (ex vivo gene therapy),
local or systemic growth factor production could be optimized for dose and duration to provide a robust and sustained osteoinductive stimulus.
The aim of this literature review is to describe the general principles of gene transfer and to summarize recent advances in their application to the orthopaedic field.