Pharmaceutical Approach to Somatic Gene Therapy

Overview

Journal Pharm Res

Specialties Pharmacology
Pharmacy

Date 1996 Nov 1

PMID 8956323

Citations 18

Authors

F D Ledley

Affiliations

Soon will be listed here.

Abstract

The pharmaceutical approach to somatic gene therapy is based on consideration of a gene as a chemical entity with specific physical, chemical and colloidal properties. The genes that are required for gene therapy are large molecules (> 1 x 10(6) Daltons, > 100 nm diameter) with a net negative charge that prevents diffusion through biological barriers such as an intact endothelium, the plasma membrane or the nuclear membrane. New methods for gene therapy are based on increasing knowledge of the pathways by which DNA may be internalized into cells and traffic to the nucleus, pharmaceutical experience with particulate drug delivery systems, and the ability to control gene expression with recombined genetic elements. This article reviews two themes in the development of gene therapies: first, the current approaches involving the administration of cells, viruses and plasmid DNA; second, the emerging pharmaceutical approach to gene therapy based on the pharmaceutical characteristics of DNA itself and methods for advanced drug delivery.

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References

Liu Y, Liggitt D, Zhong W, Tu G, Gaensler K, Debs R . Cationic liposome-mediated intravenous gene delivery. J Biol Chem. 1995; 270(42):24864-70. DOI: 10.1074/jbc.270.42.24864. View

Pinnaduwage P, Schmitt L, Huang L . Use of a quaternary ammonium detergent in liposome mediated DNA transfection of mouse L-cells. Biochim Biophys Acta. 1989; 985(1):33-7. DOI: 10.1016/0005-2736(89)90099-0. View

Zabner J, Fasbender A, Moninger T, Poellinger K, Welsh M . Cellular and molecular barriers to gene transfer by a cationic lipid. J Biol Chem. 1995; 270(32):18997-9007. DOI: 10.1074/jbc.270.32.18997. View

Loeffler J, Behr J . Gene transfer into primary and established mammalian cell lines with lipopolyamine-coated DNA. Methods Enzymol. 1993; 217:599-618. DOI: 10.1016/0076-6879(93)17091-i. View

Gao X, Huang L . A novel cationic liposome reagent for efficient transfection of mammalian cells. Biochem Biophys Res Commun. 1991; 179(1):280-5. DOI: 10.1016/0006-291x(91)91366-k. View

Goldfarb M, Weinberg R . Generation of novel, biologically active Harvey sarcoma viruses via apparent illegitimate recombination. J Virol. 1981; 38(1):136-50. PMC: 171133. DOI: 10.1128/JVI.38.1.136-150.1981. View

Remy J, Kichler A, Mordvinov V, Schuber F, Behr J . Targeted gene transfer into hepatoma cells with lipopolyamine-condensed DNA particles presenting galactose ligands: a stage toward artificial viruses. Proc Natl Acad Sci U S A. 1995; 92(5):1744-8. PMC: 42596. DOI: 10.1073/pnas.92.5.1744. View

Legendre J, Szoka Jr F . Cyclic amphipathic peptide-DNA complexes mediate high-efficiency transfection of adherent mammalian cells. Proc Natl Acad Sci U S A. 1993; 90(3):893-7. PMC: 45776. DOI: 10.1073/pnas.90.3.893. View

Wilson J, Grossman M, Cabrera J, Wu C, Wu G . A novel mechanism for achieving transgene persistence in vivo after somatic gene transfer into hepatocytes. J Biol Chem. 1992; 267(16):11483-9. View

10.

Wagner E, Plank C, Zatloukal K, Cotten M, Birnstiel M . Influenza virus hemagglutinin HA-2 N-terminal fusogenic peptides augment gene transfer by transferrin-polylysine-DNA complexes: toward a synthetic virus-like gene-transfer vehicle. Proc Natl Acad Sci U S A. 1992; 89(17):7934-8. PMC: 49829. DOI: 10.1073/pnas.89.17.7934. View

11.

Cotten M, Wagner E, Zatloukal K, Phillips S, Curiel D, Birnstiel M . High-efficiency receptor-mediated delivery of small and large (48 kilobase gene constructs using the endosome-disruption activity of defective or chemically inactivated adenovirus particles. Proc Natl Acad Sci U S A. 1992; 89(13):6094-8. PMC: 49444. DOI: 10.1073/pnas.89.13.6094. View

12.

Wickham T, Filardo E, Cheresh D, Nemerow G . Integrin alpha v beta 5 selectively promotes adenovirus mediated cell membrane permeabilization. J Cell Biol. 1994; 127(1):257-64. PMC: 2120185. DOI: 10.1083/jcb.127.1.257. View

13.

Yoshimura K, Rosenfeld M, Seth P, Crystal R . Adenovirus-mediated augmentation of cell transfection with unmodified plasmid vectors. J Biol Chem. 1993; 268(4):2300-3. View

14.

Wolff J, Williams P, Acsadi G, Jiao S, Jani A, Chong W . Conditions affecting direct gene transfer into rodent muscle in vivo. Biotechniques. 1991; 11(4):474-85. View

15.

Wu C, Wilson J, Wu G . Targeting genes: delivery and persistent expression of a foreign gene driven by mammalian regulatory elements in vivo. J Biol Chem. 1989; 264(29):16985-7. View

16.

Cornetta K, Moen R, Culver K, Morgan R, McLachlin J, Sturm S . Amphotropic murine leukemia retrovirus is not an acute pathogen for primates. Hum Gene Ther. 1990; 1(1):15-30. DOI: 10.1089/hum.1990.1.1-15. View

17.

Baatz J, Bruno M, Ciraolo P, Glasser S, Stripp B, Smyth K . Utilization of modified surfactant-associated protein B for delivery of DNA to airway cells in culture. Proc Natl Acad Sci U S A. 1994; 91(7):2547-51. PMC: 43406. DOI: 10.1073/pnas.91.7.2547. View

18.

Vahlsing H, Yankauckas M, Sawdey M, Gromkowski S, Manthorpe M . Immunization with plasmid DNA using a pneumatic gun. J Immunol Methods. 1994; 175(1):11-22. DOI: 10.1016/0022-1759(94)90327-1. View

19.

Losordo D, Pickering J, Takeshita S, Leclerc G, Gal D, Weir L . Use of the rabbit ear artery to serially assess foreign protein secretion after site-specific arterial gene transfer in vivo. Evidence that anatomic identification of successful gene transfer may underestimate the potential magnitude of transgene.... Circulation. 1994; 89(2):785-92. DOI: 10.1161/01.cir.89.2.785. View

20.

Chapman G, Lim C, Gammon R, Culp S, Desper J, Bauman R . Gene transfer into coronary arteries of intact animals with a percutaneous balloon catheter. Circ Res. 1992; 71(1):27-33. DOI: 10.1161/01.res.71.1.27. View