Realizing the promise of gene therapy through collaboration and partnering: Pfizer’s view | DuchenneXchange

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Realizing the promise of gene therapy through collaboration and partnering: Pfizer’s view

key information

source: Pfizer

summary/abstract:

Gene therapy is a promising approach to altering the genetic composition of cells as a way to correct disease causing mutations or to express proteins or RNA molecules that confer a therapeutic benefit. The concept of gene therapy is straightforward: deliver nucleic acids to target cells to alter their function in a beneficial manner. Moving from concept to reality, however, is a complex process comprised of multiple steps and components, including systems for getting nucleic acids into target cells, DNA regulatory elements that control the amount, location and duration of gene expression, and production of proteins with appropriate activity to alter cellular function in the desired manner. 

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