Guest Open Access Plus | Free Content | About | Sign in | New Users: Sign up | Mark List  

Current Gene Therapy

Volume 11 Issue 3
ISSN: 1566-5232
eISSN: 1875-5631

 

   All Titles

  Current Advances in Retroviral Gene Therapy
  pp.218-228 (11) Authors: Youngsuk Yi, Moon Jong Noh, Kwan Hee Lee
 
 
      Abstract

There have been major changes since the incidents of leukemia development in X-SCID patients after the treatments using retroviral gene therapy. Due to the risk of oncogenesis caused by retroviral insertional activation of host genes, most of the efforts focused on the lentiviral therapies. However, a relative clonal dominance was detected in a patient with β-thalassemia Major, two years after the subject received genetically modified hematopoietic stem cells using lentiviral vectors. This disappointing result of the recent clinical trial using lentiviral vector tells us that the current and most advanced vector systems does not have enough safety. In this review, various safety features that have been tried for the retroviral gene therapy are introduced and the possible new ways of improvements are discussed. Additional feature of chromatin insulators, co-transduction of a suicidal gene under the control of an inducible promoter, conditional expression of the transgene only in appropriate target cells, targeted transduction, cell type-specific expression, targeted local administration, splitting of the viral genome, and site specific insertion of retroviral vector are discussed here.

 
  Keywords: Retroviral, Gene, Therapy, Safety, Insertional, Targeted, Vectors, Insulator, transgene, target cells
  Affiliation: TissueGene, Inc. 9605 Medical Center Dr., Suite 200, Rockville, MD 20850, USA.
 
  Key: New Content Free Content Open Access Plus Subscribed Content

Bentham Science Publishers
www.benthamscience.com

 

  Copyright © 1994 - 2014   Bentham Science Publishers