Nonviral gene therapy: promises and challenges

The last 10 years have seen substantial progress in the development and application of nonviral vectors in gene therapy. However, many problems remain to be resolved before nonviral gene therapy can become a standard clinical practice. This review highlights the major breakthroughs in this field. The problems and future research directions are also discussed.

[1]  Simon C Watkins,et al.  Effect of immune response on gene transfer to the lung via systemic administration of cationic lipidic vectors. , 1999, American journal of physiology. Lung cellular and molecular physiology.

[2]  Krishnendu Roy,et al.  Oral gene delivery with chitosan–DNA nanoparticles generates immunologic protection in a murine model of peanut allergy , 1999, Nature Medicine.

[3]  Scott L. Diamond,et al.  Nuclear targeting peptide scaffolds for lipofection of nondividing mammalian cells , 1999, Nature Biotechnology.

[4]  Leaf Huang,et al.  Potentiation of cationic liposome-mediated gene delivery by polycations. , 1996, Biochemistry.

[5]  C. Harding,et al.  CpG Oligodeoxynucleotides Act as Adjuvants that Switch on T Helper 1 (Th1) Immunity , 1997, The Journal of experimental medicine.

[6]  M. Rols,et al.  In vivo electrically mediated protein and gene transfer in murine melanoma , 1998, Nature Biotechnology.

[7]  R. Mumper,et al.  Systemic effect of human growth hormone after intramuscular injection of a single dose of a muscle-specific gene medicine. , 1998, Human gene therapy.

[8]  Simon C Watkins,et al.  DNA–based immunization by in vivo transfection of dendritic cells , 1996, Nature Medicine.

[9]  M. Stedman,et al.  Contribution of plasmid DNA to inflammation in the lung after administration of cationic lipid:pDNA complexes. , 1999, Human gene therapy.

[10]  N. Yang,et al.  In vivo and in vitro gene transfer to mammalian somatic cells by particle bombardment. , 1990, Proceedings of the National Academy of Sciences of the United States of America.

[11]  R. Horst,et al.  Ca2+-ATPases and their expression in the mammary gland of pregnant and lactating rats. , 1999, American journal of physiology. Cell physiology.

[12]  R. Scheule,et al.  Detailed analysis of structures and formulations of cationic lipids for efficient gene transfer to the lung. , 1996, Human gene therapy.

[13]  Dexi Liu,et al.  Hydrodynamics-based transfection in animals by systemic administration of plasmid DNA , 1999, Gene Therapy.

[14]  Simon C Watkins,et al.  Dynamic changes in the characteristics of cationic lipidic vectors after exposure to mouse serum: implications for intravenous lipofection , 1999, Gene Therapy.

[15]  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.

[16]  D. Scherman,et al.  A versatile vector for gene and oligonucleotide transfer into cells in culture and in vivo: polyethylenimine. , 1995, Proceedings of the National Academy of Sciences of the United States of America.

[17]  G. Bishop,et al.  CpG motifs in bacterial DNA trigger direct B-cell activation , 1995, Nature.

[18]  J. Marshall,et al.  A novel cationic lipid greatly enhances plasmid DNA delivery and expression in mouse lung. , 1996, Proceedings of the National Academy of Sciences of the United States of America.

[19]  G. Acsadi,et al.  Direct gene transfer into mouse muscle in vivo. , 1990, Science.

[20]  J. Ulmer,et al.  Heterologous protection against influenza by injection of DNA encoding a viral protein. , 1993, Science.

[21]  S. Durham,et al.  Liposome-mediated CFTR gene transfer to the nasal epithelium of patients with cystic fibrosis , 1995, Nature Medicine.

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

[23]  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.

[24]  M. Rice,et al.  Correction of the Mutation Responsible for Sickle Cell Anemia by an RNA-DNA Oligonucleotide , 1996, Science.

[25]  H. D. Liggitt,et al.  Factors influencing the efficiency of cationic liposome-mediated intravenous gene delivery , 1997, Nature Biotechnology.

[26]  C. Steer,et al.  Correction of the UDP-glucuronosyltransferase gene defect in the gunn rat model of crigler-najjar syndrome type I with a chimeric oligonucleotide. , 1999, Proceedings of the National Academy of Sciences of the United States of America.

[27]  R. Cortese,et al.  Efficient and regulated erythropoietin production by naked DNA injection and muscle electroporation. , 1999, Proceedings of the National Academy of Sciences of the United States of America.