Non-viral gene delivery with cationic liposome-DNA complexes.

A large amount of research activity worldwide is currently directed towards developing lipid- or polymer-based, non-viral gene vectors for therapeutic applications. This strong interest is motivated by their low toxicity, ease of production, ability to transfer large pieces of DNA into cells, and lack of immunogenic protein components. Cationic liposomes (CLs) are one of the most powerful non-viral vectors. In fact, CL-based vectors are among the prevalent synthetic carriers of nucleic acids currently used in human clinical gene therapy trials as well as in cell transfection applications for biological research. Our understanding of the mechanisms of action of CL-DNA complexes is still in its infancy. However, the relevance of a few crucial parameters, such as the lipid/DNA charge ratio (rho(chg)) and the membrane charge density of lamellar complexes (sigma(M)), is well established. To arrive at true comparisons of lipid performance, one must optimize both these parameters using a reproducible, reliable transfection assay. In this chapter, we aim to provide the reader with detailed procedures for liposome formation and transfection. It is our hope that the use of such optimized protocols will improve the comparability of transfection data obtained with novel lipids.

[1]  Joachim O. Rädler,et al.  Structure of DNA-Cationic Liposome Complexes: DNA Intercalation in Multilamellar Membranes in Distinct Interhelical Packing Regimes , 1997, Science.

[2]  J. Northrop,et al.  Lipofection: a highly efficient, lipid-mediated DNA-transfection procedure. , 1987, Proceedings of the National Academy of Sciences of the United States of America.

[3]  A. Lin,et al.  Structure and Structure—Function Studies of Lipid/Plasmid DNA Complexes , 2000, Journal of drug targeting.

[4]  D. Lasič,et al.  Liposomes: From Physics to Applications , 1993 .

[5]  E. Nabel,et al.  Direct gene transfer with DNA-liposome complexes in melanoma: expression, biologic activity, and lack of toxicity in humans. , 1993, Proceedings of the National Academy of Sciences of the United States of America.

[6]  F. Deist,et al.  Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease. , 2000, Science.

[7]  S W Hui,et al.  The role of helper lipids in cationic liposome-mediated gene transfer. , 1996, Biophysical journal.

[8]  Robijn Bruinsma,et al.  Electrostatics of DNA-cationic lipid complexes: isoelectric instability , 1998 .

[9]  Nathan F. Bouxsein,et al.  Dendritic cationic lipids with highly charged headgroups for efficient gene delivery. , 2006, Bioconjugate chemistry.

[10]  N. M. Rao,et al.  Cationic lipids for gene delivery in vitro and in vivo , 2006 .

[11]  E. Marshall.  Gene Therapy on Trial , 2000, Science.

[12]  R. Podgornik,et al.  The Structure of DNA−Liposome Complexes , 1997 .

[13]  J. Wixon,et al.  Gene therapy clinical trials worldwide 1989–2004—an overview , 2004, The journal of gene medicine.

[14]  Gerald S. Manning,et al.  Limiting Laws and Counterion Condensation in Polyelectrolyte Solutions I. Colligative Properties , 1969 .

[15]  B. Zimm,et al.  Distribution of counterions around a cylindrical polyelectrolyte and manning's condensation theory , 1984 .

[16]  R. Ian Freshney,et al.  Culture of Animal Cells , 1983 .

[17]  K. Ewert,et al.  Cationic lipid–DNA complexes for non-viral gene therapy: relating supramolecular structures to cellular pathways , 2005, Expert opinion on biological therapy.

[18]  M. Hung,et al.  Non-viral vectors for gene therapy , 2005 .

[19]  W. Gelbart,et al.  Structure, stability, and thermodynamics of lamellar DNA-lipid complexes. , 1998, Biophysical journal.

[20]  K. Ewert,et al.  Cationic lipid-DNA complexes for gene therapy: understanding the relationship between complex structure and gene delivery pathways at the molecular level. , 2004, Current medicinal chemistry.

[21]  B. Issell,et al.  Phase 1 Trial of Recombinant Human Interleukin-1β (rhIL-1β), Carboplatin, and Etoposide in Patients with Solid Cancers: Southwest Oncology Group Study 8940 , 1997 .

[22]  H. Farhood,et al.  The role of dioleoyl phosphatidylethanolamine in cationic liposome mediated gene transfer. , 1995, Biochimica et biophysica acta.

[23]  Christof von Kalle,et al.  A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency. , 2003, The New England journal of medicine.

[24]  H. Willard,et al.  Formation of de novo centromeres and construction of first-generation human artificial microchromosomes , 1997, Nature Genetics.

[25]  T. Salditt,et al.  Self-assembled DNA–cationic-lipid complexes: Two-dimensional smectic ordering, correlations, and interactions , 1998 .

[26]  Ayesha Ahmad,et al.  Three-dimensional imaging of lipid gene-carriers: membrane charge density controls universal transfection behavior in lamellar cationic liposome-DNA complexes. , 2003, Biophysical journal.

[27]  Cameron S. Osborne,et al.  LMO2-Associated Clonal T Cell Proliferation in Two Patients after Gene Therapy for SCID-X1 , 2003, Science.

[28]  T Friedmann,et al.  Overcoming the obstacles to gene therapy. , 1997, Scientific American.

[29]  K. Ewert,et al.  Efficient synthesis and cell-transfection properties of a new multivalent cationic lipid for nonviral gene delivery. , 2002, Journal of medicinal chemistry.

[30]  Nathan F. Bouxsein,et al.  A columnar phase of dendritic lipid-based cationic liposome-DNA complexes for gene delivery: hexagonally ordered cylindrical micelles embedded in a DNA honeycomb lattice. , 2006, Journal of the American Chemical Society.

[31]  Adam Bagg,et al.  Fatal systemic inflammatory response syndrome in a ornithine transcarbamylase deficient patient following adenoviral gene transfer. , 2003, Molecular genetics and metabolism.

[32]  S. W. Kim,et al.  Pharmaceutical Perspectives of Nucleic Acid-Based Therapeutics , 2002 .

[33]  T. Salditt,et al.  Phase diagram, stability, and overcharging of lamellar cationic lipid-DNA self-assembled complexes. , 1999, Biophysical journal.

[34]  T Salditt,et al.  An inverted hexagonal phase of cationic liposome-DNA complexes related to DNA release and delivery. , 1998, Science.

[35]  K. Ewert,et al.  New multivalent cationic lipids reveal bell curve for transfection efficiency versus membrane charge density: lipid–DNA complexes for gene delivery , 2005, The journal of gene medicine.

[36]  Dan Ferber,et al.  Safer and Virus-Free? , 2001, Science.

[37]  T. Niidome,et al.  Gene Therapy Progress and Prospects: Nonviral vectors , 2002, Gene Therapy.

[38]  Luigi Naldini,et al.  Viral vectors for gene therapy: the art of turning infectious agents into vehicles of therapeutics , 2001, Nature Medicine.

[39]  Andrew D. Miller.  Cationic Liposomes for Gene Therapy , 1998 .

[40]  Kai Ewert,et al.  Lipoplex structures and their distinct cellular pathways. , 2005, Advances in genetics.