Viral Vectors for Gene Therapy: Translational and Clinical Outlook.

  title={Viral Vectors for Gene Therapy: Translational and Clinical Outlook.},
  author={Melissa Kotterman and Thomas W. Chalberg and David V. Schaffer},
  journal={Annual review of biomedical engineering},
In a range of human trials, viral vectors have emerged as safe and effective delivery vehicles for clinical gene therapy, particularly for monogenic recessive disorders, but there has also been early work on some idiopathic diseases. These successes have been enabled by research and development efforts focusing on vectors that combine low genotoxicity and immunogenicity with highly efficient delivery, including vehicles based on adeno-associated virus and lentivirus, which are increasingly… 

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