TY - JOUR
T1 - Gene delivery by liposomes
AU - Nordling-David, Mirjam M.
AU - Golomb, Gershon
PY - 2013
Y1 - 2013
N2 - In gene therapy, genetic materials, such as plasmid DNA, antisense oligonucleotides (asODNs), and small interfering RNA (siRNA), can be used to treat or prevent disease. This includes replacing a mutated gene, inactivating a mutated gene, or introducing a new gene. Although gene therapy is a promising treatment option for a number of diseases (including inherited disorders, some types of cancer, and certain viral infections), successful gene therapy is hampered by the lack of effective delivery systems (viral and nonviral). There are several nonviral gene carriers, such as lipids, polymers, and peptides, that can be used for this purpose. Liposomal delivery of nucleic acids, such as plasmid DNA, asODNs, and siRNAs, represents a very promising nanocarrier system that is relatively safe and effective in delivering genes to targeted locations in the body. Lipoidbased delivery systems have also been shown to be stable in serum and plasma, have improved biodistribution, prolonged circulation half-life, and enhanced target tissue selectivity. The most common lipids used in liposomes are cationic lipids, which facilitate binding between their positively charged head group(s) to negatively charged nucleic acids. This review is focused on the most common methods of lipoid-based nanocarriers of nucleic acids for gene therapy in vivo.
AB - In gene therapy, genetic materials, such as plasmid DNA, antisense oligonucleotides (asODNs), and small interfering RNA (siRNA), can be used to treat or prevent disease. This includes replacing a mutated gene, inactivating a mutated gene, or introducing a new gene. Although gene therapy is a promising treatment option for a number of diseases (including inherited disorders, some types of cancer, and certain viral infections), successful gene therapy is hampered by the lack of effective delivery systems (viral and nonviral). There are several nonviral gene carriers, such as lipids, polymers, and peptides, that can be used for this purpose. Liposomal delivery of nucleic acids, such as plasmid DNA, asODNs, and siRNAs, represents a very promising nanocarrier system that is relatively safe and effective in delivering genes to targeted locations in the body. Lipoidbased delivery systems have also been shown to be stable in serum and plasma, have improved biodistribution, prolonged circulation half-life, and enhanced target tissue selectivity. The most common lipids used in liposomes are cationic lipids, which facilitate binding between their positively charged head group(s) to negatively charged nucleic acids. This review is focused on the most common methods of lipoid-based nanocarriers of nucleic acids for gene therapy in vivo.
KW - Drug delivery
KW - Gene technology
KW - Liposomes
KW - Nonviral gene delivery
KW - Nucleic acids
UR - http://www.scopus.com/inward/record.url?scp=84894634150&partnerID=8YFLogxK
U2 - 10.1002/ijch.201300055
DO - 10.1002/ijch.201300055
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AN - SCOPUS:84894634150
SN - 0021-2148
VL - 53
SP - 737
EP - 747
JO - Israel Journal of Chemistry
JF - Israel Journal of Chemistry
IS - 9-10
ER -