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Advances in gene therapy for rare diseases using viral vectors

  • LIN Yanni ,
  • LI Qiushi ,
  • WU Yao ,
  • LIN Jun
  • 1. Cure Genetics Co. Ltd., Suzhou 215028, China;
    2. The First Affiliated Hospital of Soochow University, Suzhou 215006, China

Received date: 2017-06-20

  Revised date: 2017-07-31

  Online published: 2017-08-26


A large part of rare diseases are caused by genetic factors, making them difficult to be cured using the conventional small or large molecular drugs. Nevertheless, the gene therapy could potentially correct such absence or abnormality of body functions caused by certain genetic disorders, thus bringing better quality of life to those rare-disease patients. Currently, many clinical trials of gene therapies are carried out, and some gene therapies have already been available on the market. The viral vectors are commonly used for the gene delivery. In this paper, a variety of viral vectors for the clinical gene delivery are discussed, including the adeno-associated virus (AAV), the retrovirus, and the lentivirus, focusing on their researches, applications, and developments in clinical trials of rare diseases. Furthermore, the advantages and disadvantages of the viral vectors are evaluated, and the potential applications, as well as the research directions of gene therapies are suggested.

Cite this article

LIN Yanni , LI Qiushi , WU Yao , LIN Jun . Advances in gene therapy for rare diseases using viral vectors[J]. Science & Technology Review, 2017 , 35(16) : 58 -63 . DOI: 10.3981/j.issn.1000-7857.2017.16.008


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