Open AccessGene Transfer Technology in Therapy: Current Applications and Future Goals
Author(s) -
Romano Gaetano,
Pacilio Carmen,
Giordano Antonio
Publication year - 1999
Publication title -
stem cells
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 2.159
H-Index - 229
eISSN - 1549-4918
pISSN - 1066-5099
DOI - 10.1002/stem.170191
Subject(s) - genetic enhancement , gene transfer , clinical trial , biology , gene delivery , viral vector , psychological intervention , intensive care medicine , vector (molecular biology) , bioinformatics , computational biology , gene , medicine , genetics , psychiatry , recombinant dna
Abstract Gene therapy has attracted much interest since the first submissions of phase I clinical trials in the early 1990s, for the treatment of inherited genetic diseases. Preliminary results were very encouraging and prompted many investigators to submit protocols for phase I and phase II clinical trials for the treatment of inherited genetic diseases and cancer. The possible application of gene transfer technology to treat AIDS, cardiopathies, and neurologic diseases is under evaluation. Some viral vectors have already been used to deliver HIV‐1 subunits to immunize volunteers who are participating in the AIDS vaccine programs in the USA. However, gene delivery systems still need to be optimized in order to achieve effective therapeutic interventions. The purpose of this review is to summarize the latest achievements in improving gene delivery systems, their current application in preclinical studies and in therapy, and the most pressing issues that must be addressed in the area of vector design.