Development of non-viral vectors for systemic gene delivery.
One of the major challenges for gene therapy is systemic delivery of a nucleic acid directly into an affected tissue. This requires developing a vehicle which is able to protect the nucleic acid from degradation, while delivering the gene of interest to the specific tissue and specific subcellular compartment. In this review, we summarize some of the recent advances in new non-viral delivery systems for systemic administration. Two types of gene delivery systems are described: (i) LPD1 (cationic liposome-entrapped, polycation-condensed DNA, type 1), and (ii) retention-time mediated naked DNA delivery. Hypothesized mechanisms for these systemic gene transfers are also discussed.
Liu
Feng
F
Center for Pharmacogenetics, School of Pharmacy, 633 Salk Hall, University of Pittsburgh, Pittsburgh, PA 15213, USA.
Huang
Leaf
L
D000818:Animals; D004247:DNA / Q000008:administration & dosage; D018014:Gene Transfer Techniques; D015316:Genetic Therapy; D005822:Genetic Vectors; D006801:Humans; D008081:Liposomes
D008081:Liposomes; D004247:DNA