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Adv Pharm Bull. 2013;3(2): 249-255.
doi: 10.5681/apb.2013.041
PMID: 24312844
PMCID: PMC3848228
Scopus ID: 84885753625
  Abstract View: 1278
  PDF Download: 924

Original Research

Gene Therapy, Early Promises, Subsequent Problems, and Recent Breakthroughs

Saeideh Razi Soofiyani, Behzad Baradaran*, Farzaneh Lotfipour, Tohid Kazemi, Leila Mohammadnejad
*Corresponding Author: Email: behzad_im@yahoo.com

Abstract

Gene therapy is one of the most attractive fields in medicine. The concept of gene delivery to tissues for clinical applications has been discussed around half a century, but scientist’s ability to manipulate genetic material via recombinant DNA technology made this purpose to reality. Various approaches, such as viral and non-viral vectors and physical methods, have been developed to make gene delivery safer and more efficient. While gene therapy initially conceived as a way to treat life-threatening disorders (inborn errors, cancers) refractory to conventional treatment, to date gene therapy is considered for many non–life-threatening conditions including those adversely influence on a patient’s quality of life. Gene therapy has made significant progress, including tangible success, although much slower than was initially predicted. Although, gene therapies still at a fairly primitive stage, it is firmly science based. There is justifiable hope that with enhanced pathobiological understanding and biotechnological improvements, gene therapy will be a standard part of clinical practice within 20 years.
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Submitted: 13 Feb 2013
Revision: 08 Apr 2013
ePublished: 20 Aug 2013
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