Open AccessThe challenge of gene therapy and DNA delivery
Author(s) -
Phillips Anthony J.
Publication year - 2001
Publication title -
journal of pharmacy and pharmacology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 0.745
H-Index - 118
eISSN - 2042-7158
pISSN - 0022-3573
DOI - 10.1211/0022357011776603
Subject(s) - genetic enhancement , gene transfer , optimism , gene delivery , gene , medicine , bioinformatics , intensive care medicine , biology , computational biology , genetics , psychology , psychotherapist
It is 10 years since the first gene therapeutic was administered to patients. Since that time approximately 3500 patients have been treated and although some evidence of gene transfer has been seen it has generally been inadequate for a meaningful clinical response. The major challenges have been delivery of DNA to the target cells and duration of expression. A variety of gene transfer systems have been evaluated and these are summarized with their advantages and disadvantages. Encouraging data have been obtained recentlyby a number of workers and there is some optimism that the enormous potential of gene therapy will be realized in the second decade of its development.