Gene Therapy That Works

@article{Verma2013GeneTT,
  title={Gene Therapy That Works},
  author={Inder M. Verma},
  journal={Science},
  year={2013},
  volume={341},
  pages={853 - 855}
}
  • I. Verma
  • Published 23 August 2013
  • Biology, Medicine
  • Science
Gene therapy trials show a beneficial effect in children suffering from a neurodegenerative disorder or an immunodeficiency disease. [Also see Research Articles by Biffi et al. and Aiuti et al.] The concept of gene therapy is disarmingly simple: Introduce a healthy gene in a patient and its product should alleviate the defect caused by a faulty gene or slow the progression of disease (1). Why then, over the past three decades, have there been so few clinical successes in treating patients with… 
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