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May 14, 1993Science1,765 citations

The Basic Science of Gene Therapy

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RMRichard C. Mulligan

Key Points

  • To review the scientific foundations of gene-based therapies and evaluate the remaining technical challenges hindering clinical translation.
  • Reviewed decades of technological progress in gene transfer to mammalian cells.
  • Assessed technical bottlenecks related to delivery vectors and cell transplantation methods.
  • Substantial progress in gene delivery methods has established the feasibility of mammalian cell genetic modification.
  • Clinical safety and efficacy remain limited by key technical hurdles, particularly efficient gene transfer mechanisms and effective cell transplantation strategies.

Abstract

The development over the past decade of methods for delivering genes to mammalian cells has stimulated great interest in the possibility of treating human disease by gene-based therapies. However, despite substantial progress, a number of key technical issues need to be resolved before gene therapy can be safely and effectively applied in the clinic. Future technological developments, particularly in the areas of gene delivery and cell transplantation, will be critical for the successful practice of gene therapy.

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Cite This Study

Richard C. Mulligan (1993) studied this question.

synapsesocial.com/papers/6a090a0936c557a7e273b71chttps://doi.org/10.1126/science.8493530
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