Controlled release systems for DNA delivery.

Adapting controlled release technologies to the delivery of DNA has the potential to overcome extracellular barriers that limit gene therapy. Controlled release systems can enhance gene delivery and increase the extent and duration of transgene expression relative to more traditional delivery methods (e.g., injection). These systems typically deliver vectors locally, which can avoid distribution to distant tissues, decrease toxicity to nontarget cells, and reduce the immune response to the vector. Delivery vehicles for controlled release are fabricated from natural and synthetic polymers, which function either by releasing the vector into the local tissue environment or by maintaining the vector at the polymer surface. Vector release or binding is regulated by the effective affinity of the vector for the polymer, which depends upon the strength of molecular interactions. These interactions occur through nonspecific binding based on vector and polymer composition or through the incorporation of complementary binding sites (e.g., biotin-avidin). This review examines the delivery of nonviral and viral vectors from natural and synthetic polymers and presents opportunities for continuing developments to increase their applicability.

[1]  D. Putnam,et al.  PLGA microspheres containing plasmid DNA: preservation of supercoiled DNA via cryopreparation and carbohydrate stabilization. , 1999, Journal of pharmaceutical sciences.

[2]  M. L. La Rotonda,et al.  Poly(lactide-co-glycolide) microspheres for the controlled release of oligonucleotide/polyethylenimine complexes. , 2002, Journal of pharmaceutical sciences.

[3]  Kristi S. Anseth,et al.  Gene Delivery in Tissue Engineering: A Photopolymer Platform to Coencapsulate Cells and Plasmid DNA , 2003, Pharmaceutical Research.

[4]  S. Andreadis,et al.  High Efficiencies of Gene Transfer with Immobilized Recombinant Retrovirus: Kinetics and Optimization , 2001, Biotechnology progress.

[5]  R. Barr,et al.  Electromobility of plasmid DNA in tumor tissues during electric field-mediated gene delivery , 2002, Gene Therapy.

[6]  M. Spector,et al.  Gene delivery to cartilage defects using coagulated bone marrow aspirate , 2004, Gene Therapy.

[7]  P. Hornsby,et al.  Transfection by Polyethyleneimine-Coated Microspheres , 2001, Journal of drug targeting.

[8]  H. HogenEsch,et al.  Encapsulation of recombinant adenovirus into alginate microspheres circumvents vector-specific immune response , 2002, Gene Therapy.

[9]  Jayanth Panyam,et al.  Biodegradable nanoparticles for drug and gene delivery to cells and tissue. , 2003, Advanced drug delivery reviews.

[10]  M. Barry,et al.  Metabolic biotinylation of recombinant proteins in mammalian cells and in mice. , 2000, Molecular therapy : the journal of the American Society of Gene Therapy.

[11]  C. Plank,et al.  Nonviral vector loaded collagen sponges for sustained gene delivery in vitro and in vivo , 2002, The journal of gene medicine.

[12]  M. Barry,et al.  Metabolic biotinylation of secreted and cell surface proteins from mammalian cells. , 2001, Biochemical and biophysical research communications.

[13]  Y. Tabata,et al.  Local delivery of matrix metalloproteinase gene prevents the onset of renal sclerosis in streptozotocin-induced diabetic mice. , 2003, Tissue engineering.

[14]  Weiliam Chen,et al.  Characterization of DNA-hyaluronan matrix for sustained gene transfer. , 2003, Journal of controlled release : official journal of the Controlled Release Society.

[15]  Y. Capan,et al.  Influence of formulation parameters on the characteristics of poly(D, L-lactide-co-glycolide) microspheres containing poly(L-lysine) complexed plasmid DNA. , 1999, Journal of controlled release : official journal of the Controlled Release Society.

[16]  W. Anderson,et al.  Human gene therapy. , 1992, Science.

[17]  Cato T Laurencin,et al.  Development of a calcium phosphate co-precipitate/poly(lactide-co-glycolide) DNA delivery system: release kinetics and cellular transfection studies. , 2004, Biomaterials.

[18]  N. Weiner,et al.  Application of membrane-based dendrimer/DNA complexes for solid phase transfection in vitro and in vivo. , 2000, Biomaterials.

[19]  C. Dinney,et al.  Repeated intravesical instillations of an adenoviral vector in patients with locally advanced bladder cancer: a phase I study of p53 gene therapy. , 2003, Journal of clinical oncology : official journal of the American Society of Clinical Oncology.

[20]  N. Rainov,et al.  Gene Therapy for Human Malignant Brain Tumors , 2003, Cancer journal.

[21]  Jeffrey Bonadio,et al.  Localized, direct plasmid gene delivery in vivo: prolonged therapy results in reproducible tissue regeneration , 1999, Nature Medicine.

[22]  S. Diamond,et al.  Endovascular Microcoil Gene Delivery Using Immobilized Anti-adenovirus Antibody for Vector Tethering , 2002, Stroke.

[23]  Y. Capan,et al.  Stability of poly(L-lysine)-complexed plasmid DNA during mechanical stress and DNase I treatment. , 1999, Pharmaceutical development and technology.

[24]  H. Cavanagh,et al.  Cell contact dependent extended release of adenovirus by microparticles in vitro. , 2001, Journal of virological methods.

[25]  Helmuth van Es,et al.  Arrayed adenoviral expression libraries for functional screening , 2002, Nature Biotechnology.

[26]  B. Olsen,et al.  Delivery of plasmid DNA to articular chondrocytes via novel collagen-glycosaminoglycan matrices. , 2002, Human gene therapy.

[27]  Davidson,et al.  Extended release of adenovirus from polymer microspheres: potential use in gene therapy for brain tumors. , 1997, Advanced drug delivery reviews.

[28]  C. Bertozzi,et al.  Engineering Novel Cell Surface Receptors for Virus-mediated Gene Transfer* , 1999, The Journal of Biological Chemistry.

[29]  Weiliam Chen,et al.  Hyaluronan microspheres for sustained gene delivery and site-specific targeting. , 2004, Biomaterials.

[30]  K. Leong,et al.  Gene transfer by DNA-gelatin nanospheres. , 1999, Archives of biochemistry and biophysics.

[31]  H. Roehl,et al.  Phase 1 trial of FVIII gene transfer for severe hemophilia A using a retroviral construct administered by peripheral intravenous infusion. , 2003, Blood.

[32]  D. Lauffenburger,et al.  Quantitative analysis of synthetic gene delivery vector design properties. , 2001, Molecular therapy : the journal of the American Society of Gene Therapy.

[33]  G F Pierce,et al.  Collagen-embedded platelet-derived growth factor DNA plasmid promotes wound healing in a dermal ulcer model. , 2000, The Journal of surgical research.

[34]  M. Herlyn,et al.  Matrix immobilization enhances the tissue repair activity of growth factor gene therapy vectors. , 2001, Human gene therapy.

[35]  R. Levy,et al.  Gene delivery from a DNA controlled-release stent in porcine coronary arteries , 2000, Nature Biotechnology.

[36]  K. Yip,et al.  Controlled release of plasmid DNA , 1997 .

[37]  I. Alferiev,et al.  Localized gene delivery using antibody tethered adenovirus from polyurethane heart valve cusps and intra-aortic implants , 2004, Gene therapy.

[38]  W. Mark Saltzman,et al.  Enhancement of transfection by physical concentration of DNA at the cell surface , 2000, Nature Biotechnology.

[39]  J. Engh,et al.  In vivo endochondral bone formation using a bone morphogenetic protein 2 adenoviral vector. , 1999, Human gene therapy.

[40]  Jon Dobson,et al.  Improved method of recombinant AAV2 delivery for systemic targeted gene therapy. , 2002, Molecular therapy : the journal of the American Society of Gene Therapy.

[41]  F. Szoka,et al.  Robust and prolonged gene expression from injectable polymeric implants , 2002, Gene Therapy.

[42]  Scott D. Putney,et al.  Improving protein therapeutics with sustained-release formulations , 1998, Nature Biotechnology.

[43]  B. Davidson,et al.  Poly-L-lysine improves gene transfer with adenovirus formulated in PLGA microspheres , 1999, Gene Therapy.

[44]  Hassan Adwan,et al.  Delivery and expression of pDNA embedded in collagen matrices. , 2004, Journal of controlled release : official journal of the Controlled Release Society.

[45]  David M Sabatini,et al.  Cell-biological applications of transfected-cell microarrays. , 2002, Trends in cell biology.

[46]  H. Herweijer,et al.  Progress and prospects: naked DNA gene transfer and therapy , 2003, Gene Therapy.

[47]  T. Ochiya,et al.  Biomaterials for gene delivery: atelocollagen-mediated controlled release of molecular medicines. , 2001, Current gene therapy.

[48]  J. Hubbell,et al.  Development of growth factor fusion proteins for cell‐triggered drug delivery , 2001, FASEB journal : official publication of the Federation of American Societies for Experimental Biology.

[49]  W. Saltzman,et al.  Gene expression and mucosal immune responses after vaginal DNA immunization in mice using a controlled delivery matrix. , 2003, Journal of controlled release : official journal of the Controlled Release Society.

[50]  M. Herlyn,et al.  FGF2-Targeted adenovirus encoding platelet-derived growth factor-B enhances de novo tissue formation. , 2000, Molecular therapy : the journal of the American Society of Gene Therapy.

[51]  D. Kniss,et al.  Three-dimensional cell-scaffold constructs promote efficient gene transfection: implications for cell-based gene therapy. , 2001, Tissue engineering.

[52]  J. Tartaglia,et al.  Viral vector delivery in solid-state vehicles: gene expression in a murine prostate cancer model. , 2000, Journal of the National Cancer Institute.

[53]  R. Proctor.  Fibronectin: a brief overview of its structure, function, and physiology. , 1987, Reviews of infectious diseases.

[54]  W. Mark Saltzman,et al.  Building drug delivery into tissue engineering design , 2002, Nature Reviews Drug Discovery.

[55]  K. Leong,et al.  Coacervate microspheres as carriers of recombinant adenoviruses , 1999, Cancer Gene Therapy.

[56]  P. Hornsby,et al.  Transfection of Cells Mediated by Biodegradable Polymer Materials with Surface‐Bound Polyethyleneimine , 2000, Biotechnology progress.

[57]  S. Goldstein,et al.  Gene transfection using biodegradable nanospheres: results in tissue culture and a rat osteotomy model , 1999 .

[58]  Leaf Huang,et al.  Nonviral vectors in the new millennium: delivery barriers in gene transfer. , 2001, Human gene therapy.

[59]  J. L. Raina,et al.  Factors underlying spontaneous inactivation and susceptibility to neutralization of human immunodeficiency virus. , 1992, Virology.

[60]  T. Segura,et al.  Substrate-mediated DNA delivery: role of the cationic polymer structure and extent of modification. , 2003, Journal of controlled release : official journal of the Controlled Release Society.

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

[62]  L. Barrett,et al.  Sustained Effects of Gene-Activated Matrices after CNS Injury , 2001, Molecular and Cellular Neuroscience.

[63]  P. Weston,et al.  Transfection of HEK Cells via DNA-loaded PLGA and P(FASA) Nanospheres , 2002, Journal of drug targeting.

[64]  Tatiana Segura,et al.  Materials for Non-Viral Gene Delivery , 2001 .

[65]  Donald E. Chickering,et al.  Biologically erodable microspheres as potential oral drug delivery systems , 1997, Nature.

[66]  Lonnie D Shea,et al.  Drug-releasing scaffolds fabricated from drug-loaded microspheres. , 2002, Journal of biomedical materials research.

[67]  M. Benet,et al.  Stability of PEI-DNA and DOTAP-DNA complexes: effect of alkaline pH, heparin and serum. , 2001, Journal of controlled release : official journal of the Controlled Release Society.

[68]  F. Szoka,et al.  Effect of serum components on the physico-chemical properties of cationic lipid/oligonucleotide complexes and on their interactions with cells. , 1998, Biochimica et biophysica acta.

[69]  P. Robbins,et al.  Adenovirus-mediated direct gene therapy with bone morphogenetic protein-2 produces bone. , 1999, Bone.

[70]  K. Leong,et al.  DNA-polycation nanospheres as non-viral gene delivery vehicles. , 1998, Journal of controlled release : official journal of the Controlled Release Society.

[71]  O. Ludkovski,et al.  Stabilized plasmid-lipid particles for systemic gene therapy. , 2000, Gene therapy.

[72]  L. Chandler,et al.  Delivery of FGF genes to wound repair cells enhances arteriogenesis and myogenesis in skeletal muscle. , 2002, Molecular therapy : the journal of the American Society of Gene Therapy.

[73]  David J Mooney,et al.  Fabrication and in vitro testing of polymeric delivery system for condensed DNA. , 2003, Journal of biomedical materials research. Part A.

[74]  D. McDonald,et al.  Plasmid DNA adsorbed onto cationic microparticles mediates target gene expression and antigen presentation by dendritic cells , 2000, Gene Therapy.

[75]  Daniel W Pack,et al.  Microspheres for controlled release drug delivery , 2004, Expert opinion on biological therapy.

[76]  T. Ochiya,et al.  New delivery system for plasmid DNA in vivo using atelocollagen as a carrier material: the Minipellet , 1999, Nature Medicine.

[77]  Fred D. Ledley,et al.  Pharmaceutical Approach to Somatic Gene Therapy , 1996, Pharmaceutical Research.

[78]  D. Kerr,et al.  Taking Gene Therapy into the Clinic , 2003, Journal of Biomedicine and Biotechnology.

[79]  B. Davidson,et al.  Poly (lactic-glycolic) acid copolymer encapsulation of recombinant adenovirus reduces immunogenicity in vivo , 1998, Gene Therapy.

[80]  T. Schiestel,et al.  A nonviral DNA delivery system based on surface modified silica-nanoparticles can efficiently transfect cells in vitro. , 2000, Bioconjugate chemistry.

[81]  D. Sabatini,et al.  Microarrays of cells expressing defined cDNAs , 2001, Nature.

[82]  David Kerr,et al.  Clinical development of gene therapy for colorectal cancer , 2003, Nature Reviews Cancer.

[83]  W. Norde,et al.  Why proteins prefer interfaces. , 1991, Journal of biomaterials science. Polymer edition.

[84]  R. Langer,et al.  A theoretical model of erosion and macromolecular drug release from biodegrading microspheres. , 1997, Journal of pharmaceutical sciences.

[85]  Jayanth Panyam,et al.  Rapid endo‐lysosomal escape of poly(DL‐lactide‐coglycolide) nanoparticles: implications for drug and gene delivery , 2002, FASEB journal : official publication of the Federation of American Societies for Experimental Biology.

[86]  M. L. La Rotonda,et al.  Biodegradable microparticles for the controlled delivery of oligonucleotides. , 2002, International journal of pharmaceutics.

[87]  R. Levy,et al.  Gene delivery to pig coronary arteries from stents carrying antibody-tethered adenovirus. , 2002, Human gene therapy.

[88]  E. Canning,et al.  A triploblast origin for Myxozoa? , 1998, Nature.

[89]  R. Levy,et al.  Localized adenovirus gene delivery using antiviral IgG complexation , 2001, Gene Therapy.

[90]  R. Levy,et al.  Sustained delivery and expression of DNA encapsulated in polymeric nanoparticles , 2000, Gene Therapy.

[91]  Jae-Hyung Jang,et al.  Controllable delivery of non-viral DNA from porous scaffolds. , 2003, Journal of controlled release : official journal of the Controlled Release Society.

[92]  Tatiana Segura,et al.  Surface-tethered DNA complexes for enhanced gene delivery. , 2002, Bioconjugate chemistry.

[93]  Y. Tabata,et al.  Biodegradable gelatin hydrogel potentiates the angiogenic effect of fibroblast growth factor 4 plasmid in rabbit hindlimb ischemia. , 2003, Journal of the American College of Cardiology.

[94]  S. Miyatake,et al.  Osteoinduction by bone morphogenetic protein-2 via adenoviral vector under transient immunosuppression. , 2000, Biochemical and biophysical research communications.

[95]  M. Pandori,et al.  Adenovirus-microbead conjugates possess enhanced infectivity: a new strategy for localized gene delivery. , 2002, Virology.

[96]  David J. Mooney,et al.  DNA delivery from polymer matrices for tissue engineering , 1999, Nature Biotechnology.

[97]  B. Palsson,et al.  The physico-chemical factors that govern retrovirus-mediated gene transfer. , 1997, Experimental hematology.

[98]  S. Mohapatra.  Mucosal gene expression vaccine: a novel vaccine strategy for respiratory syncytial virus , 2003, The Pediatric infectious disease journal.

[99]  S. Goldstein,et al.  Stimulation of new bone formation by direct transfer of osteogenic plasmid genes. , 1996, Proceedings of the National Academy of Sciences of the United States of America.

[100]  T Ochiya,et al.  Atelocollagen-based gene transfer in cells allows high-throughput screening of gene functions. , 2001, Biochemical and biophysical research communications.

[101]  D. Mooney,et al.  Controlled delivery of inductive proteins, plasmid DNA and cells from tissue engineering matrices. , 1999, Journal of periodontal research.