Nancy Smyth Templeton

New directions in liposome gene delivery.

The history of liposomes, progress in liposome gene delivery, and future directions are discussed. Specific characteristics of liposomes and DNA:liposome complexes have been identified that are essential for optimal delivery and gene expression. Of particular interest are the requirements for increased delivery and high levels of gene expression in vivo. At present, significant efforts are focused towards achieving specific delivery and gene expression in target organs and tissues.

Cationic Liposome-Mediated Gene Delivery In vivo

Several improvements have been made in liposomal delivery, thus making this technology potentially useful for treatment of certain diseases in the clinic. Success in non-viral delivery is complicated and requires optimization of several components. These components include nucleic acid purification, plasmid design, formulation of the delivery vehicle, administration route and schedule, dosing, detection of gene expression, and others. With further improvements, broad use of non-viral delivery systems to treat human disorders should be possible.Several improvements have been made in liposomal delivery, thus making this technology potentially useful for treatment of certain diseases in the clinic. Success in non-viral delivery is complicated and requires optimization of several components. These components include nucleic acid purification, plasmid design, formulation of the delivery vehicle, administration route and schedule, dosing, detection of gene expression, and others. With further improvements, broad use of non-viral delivery systems to treat human disorders should be possible.

Myths concerning the use of cationic liposomes in vivo

Varied results have been obtained using cationic liposomes for in vivo delivery. Furthermore, optimisation of cationic liposomal complexes for in vivo applications is complicated, involving many diverse components. These components include nucleic acid purification, plasmid design, formulation of the delivery vehicle, administration route and schedule, dosing, detection of gene expression and others. Broad assumptions have frequently been made based on data obtained from focused studies using cationic liposomes. However, these assumptions do not necessarily apply to all delivery vehicles and, most likely, do not apply to many liposomal systems, when considering these other key components which influence the results obtained in vivo. Optimising all the components of the delivery system is pivotal and will allow broad use of liposomal complexes to treat or cure human diseases or disorders. This review will highlight the features of liposomes that contribute to successful delivery, gene expression and efficacy.Varied results have been obtained using cationic liposomes for in vivo delivery. Furthermore, optimisation of cationic liposomal complexes for in vivo applications is complicated, involving many diverse components. These components include nucleic acid purification, plasmid design, formulation of the delivery vehicle, administration route and schedule, dosing, detection of gene expression and others. Broad assumptions have frequently been made based on data obtained from focused studies using cationic liposomes. However, these assumptions do not necessarily apply to all delivery vehicles and, most likely, do not apply to many liposomal systems, when considering these other key components which influence the results obtained in vivo. Optimising all the components of the delivery system is pivotal and will allow broad use of liposomal complexes to treat or cure human diseases or disorders. This review will highlight the features of liposomes that contribute to successful delivery, gene expression and efficacy.

Nonviral Vectors for the Treatment of Disease

1973 B.S., City College of the City University, New York, NY 1982 M.S., Genetics and Cell Biology, University of Connecticut, Storrs, CT 1988 Ph.D., Molecular Biology and Biochemistry, Wesleyan University, Middletown, CT 1989-1991 Biotechnology Fellow, Laboratory of Pathology, National Cancer Institute, NIH, Bethesda, MD 1991-1994 Senior Staff Fellow, Molecular Hematology Branch, NHLBI, NIH, Bethesda, MD 1994-1995 Senior Scientist, Megabios Corp., Burlingame, CA 1995-1998 Scientist, NCI-FCRDC, ABL-Basic Research Program, Frederick, MD 1998-present Assistant Professor, Department of Molecular and Cellular Biology, Center for Cell and Gene Therapy, Baylor College of Medicine, Houston, TX

New Cationic Liposomes for Gene Transfer

Publisher Summary This chapter describes the role of new cationic liposomes in gene transfer. Liposomes condense nucleic acids, mixtures of nucleic acids and proteins, and viruses on the interior of bilamellar invaginated vesicles (BIVs) produced by a novel extrusion procedure. The ligands include monoclonal antibodies, Fab fragments, peptides, peptide mimetics, small molecules and drugs, proteins, and parts of proteins. Access the mobility for the conductivity calibration standard and the mobility standard using the zeta potential analyzer. If the standard is within range, proceed to assessing the liposome stock. The particle size of the complexes can also be measured. One needs to determine the size for the L300 and L500 standards using the particle size analyzer. If the standards are within range, proceed to assessing the complexes. Place 12 μl of complexes into 4 ml of sterile water in the plastic cuvette. Determine the particle size, which should be about 400 nm, and the acceptable range is between 200 and 500 nm.