Skip to main content
SpringerLink
Log in

Targeted and Sustained-Release Delivery Concepts in Gene Therapy

  • Chapter
Gene Transfer in the Cardiovascular System

Part of the book series: Developments in Cardiovascular Medicine ((DICM,volume 189))

  • 56 Accesses

Abstract

There are presently a number of applications for gene therapy, and a number of promising approaches. This chapter describes a variety of methodologies that could potentially be employed to address some of the deficiencies of current delivery vehicles employed in gene therapy. In particular, approaches that may provide for specific targeting or the sustained release of therapeutically administered genetic material are discussed.

This is a preview of subscription content, log in via an institution to check access.

Access this chapter

Log in via an institution
Chapter
USD 29.95
Price excludes VAT (USA)
  • Available as PDF
  • Read on any device
  • Instant download
  • Own it forever
eBook
USD 169.00
Price excludes VAT (USA)
  • Available as EPUB and PDF
  • Read on any device
  • Instant download
  • Own it forever
Softcover Book
USD 219.99
Price excludes VAT (USA)
  • Compact, lightweight edition
  • Dispatched in 3 to 5 business days
  • Free shipping worldwide - see info
Hardcover Book
USD 219.99
Price excludes VAT (USA)
  • Durable hardcover edition
  • Dispatched in 3 to 5 business days
  • Free shipping worldwide - see info

Tax calculation will be finalised at checkout

Purchases are for personal use only

Institutional subscriptions

Preview

Unable to display preview. Download preview PDF.

Unable to display preview. Download preview PDF.

References

  1. Hoeben RC, Valerio D, van der Eb AJ, van Ormondt H: Gene therapy for human inherited disorders: techniques and status. Critical Reviews in Oncology/Hematology 1992; 13:33–54

    Article  PubMed  CAS  Google Scholar 

  2. Morsy MA, Mitani K, Clemens P, Caskey T: Progress toward human gene therapy. JAMA 1993; 270:2338–2345

    Article  PubMed  CAS  Google Scholar 

  3. Walsh CE, Liu JM, Miller J, Nienhuis AW, Samulski RJ: Gene therapy for human hemoglobinopathies. Proc Soc Exp Biol Med 1993; 204:289–300

    PubMed  CAS  Google Scholar 

  4. Freeman SM, Zwiebel JA: Gene therapy of cancer. Cancer Investigation 1993; 11:676–688

    Article  PubMed  CAS  Google Scholar 

  5. Zwiebel JA, Su N, MacPherson A, Davis T, Ojeifo JO: The gene therapy of cancer: transgenic immunotherapy. Seminars in Hematology 1993; 30:119–129

    PubMed  CAS  Google Scholar 

  6. Chang AGY, Wu GY: Gene Therapy: applications to the treatment of gastrointestinal and liver diseases. Gastroenterology 1994; 106:1076–1084

    PubMed  CAS  Google Scholar 

  7. Ledley FD: Hepatic gene therapy: present and future. Hepatology 1993; 18:1263–1273

    Article  PubMed  CAS  Google Scholar 

  8. Suhr ST, Gage FH: Gene therapy for neurologic disease. Arch Neurol 1993; 50:1252–1268

    Article  PubMed  CAS  Google Scholar 

  9. Kennedy PGE, Steiner I: The use of herpes simplex virus vectors for gene therapy in neurological diseases. Quarterly J Med 1993; 86:697–702

    CAS  Google Scholar 

  10. Karpati G, Acsadi G: The potential for gene therapy in duchenne muscular dystrophy and other genetic muscle diseases. Muscle & Nerve 1993; 16:1141–1153

    Article  CAS  Google Scholar 

  11. Newgard CB: Cellular engineering and gene therapy strategies for insulin replacement in diabetes. Diabetes 1994; 43:341–350

    Article  PubMed  CAS  Google Scholar 

  12. Gilboa E, Smith C: Gene therapy for infectious diseases: the AIDS model. TIG 1994; 10:139–144

    Article  PubMed  CAS  Google Scholar 

  13. McCabe ERB: Clinical aplication of gene therapy: emerging opportunities and current limitations. Biochem Med Metab Biol 1993; 50:241–253

    Article  PubMed  CAS  Google Scholar 

  14. Kielian M, Jungerwirth S: Mechanisms of enveloped virus entry into cells. Mol Biol Med 1990; 7:17–31

    PubMed  CAS  Google Scholar 

  15. Choppin PW, Scheid A: The role of viral glycoproteins in adsorption, penetration, and pathogenicity of viruses. Rev Infectious Dis 1980; 2:40–61

    Article  CAS  Google Scholar 

  16. Helenius A: Unpacking the incoming influenza virus. Cell 1992; 69:577–578

    Article  PubMed  CAS  Google Scholar 

  17. Eglitis MA, Anderson WF: Retroviral vetors for introduction of genes into mammalian cells. Bio Techniques 1988; 6:608–614

    CAS  Google Scholar 

  18. Berkner KL: Development of adenovirus vectors for the expression of heterologous genes. Bio Techniques 1988; 6:616–629

    CAS  Google Scholar 

  19. Samulski RJ, Zhu X, Xiao X: Targeted integration of adeno-associated virus (AAV) into human chromosome 19. EMBO J 1991; 10:3941–3950

    PubMed  CAS  Google Scholar 

  20. Cohen-Haguenauer O: Transfer of genes, homologous recombination and genotherapy in hematology. Nouv Rev Fr Hematol 1994; 36:S3–S9

    PubMed  CAS  Google Scholar 

  21. Breakefield XO: Gene delivery into the brain using virus vectors. Nature Genet 1993; 3:187–189

    Article  PubMed  CAS  Google Scholar 

  22. Johnson P, et al.: Cytotoxicity of a replication-defective mutant of herpes simplex virus type 1. J Virol 1992; 66:2952

    PubMed  CAS  Google Scholar 

  23. Curiel DT: High-efficiency gene transfer employing adenovirus-polylysine-DNA complexes. Nat Immun 1994; 13:141–164

    PubMed  CAS  Google Scholar 

  24. Loyter A, Schangos GA, Ruddle FH: Mechanisms of DNA uptake by mammalian cells: fate of exogenously added DNA monitored by the use of fluorescent dyes. Proc Natl Acad Sci USA 1982; 79:422–426

    Article  PubMed  CAS  Google Scholar 

  25. Wolff JA, Malone RW, Williams P et al.: Direct gene transfer into mouse muscle in vivo. Science 1990; 247:1465–1468

    Article  PubMed  CAS  Google Scholar 

  26. Yang NS, Burkholder J, Roberts B, Martinell B, McCabe D: In vivo and in vitro gene transfer to mammalian somatic cells by particle bombardment. Proc Natl Acad Sci USA 1990; 87:9568–9572

    Article  PubMed  CAS  Google Scholar 

  27. Matthews KE, Mills GB, Horsfall W, Hack N, Skorecki K, Keating A: Bead transfection: rapid and efficient gene transfer into marrow stromal and other adherent mammalian cells. Experimental Hematology 1993; 21:697–702

    PubMed  CAS  Google Scholar 

  28. Mannino RJ, Gould-Fogerite S: Lipsome mediated gene tranfer. Bio Techniques 1988; 6:682–690

    CAS  Google Scholar 

  29. Akhtar S, Juliano RL: Liposome delivery of antisense oligonucleotides: adsorption and efflux charateristics of phosphorothioate oligodeoxynucleotides. J Controlled Release 1992; 22:47–56

    Article  CAS  Google Scholar 

  30. Feigner PL, Gadek TR, Holm M et al.: Lipofection: a highly efficient, lipid-mediated DNA-transfection procedure. Proc Natl Acad Sci USA 1987; 84:7413–7417

    Article  Google Scholar 

  31. Wu GY, Wu CH: Receptor-mediated gene delivery and expression in vivo. J Biol Chetn 1988; 263:14621–14624

    CAS  Google Scholar 

  32. Zenke M, Steinlein P, Wagner E, Cotten M, Beug H, Birnstiel ML: Receptor-mediated endocytosis of transferrin-polycation conjugates: an efficient way to introduce DNA into hematopoietic cells. Proc Natl Acad Sci USA 1990; 87:3655–3659

    Article  PubMed  CAS  Google Scholar 

  33. Cotten M, Langle-Rouault F, Kirlappos H et al.: Transferrin-polycation-mediated introduction of DNA into human leukemic cells: stimulation by agents that affect the survival of transfected DNA or modulate transferrin receptor levels. Proc Natl Acad Sci USA 1990; 87:4033–4037

    Article  PubMed  CAS  Google Scholar 

  34. Seth P, Fitzgerald D, Ginsberg H, Willingham M, Pastan I: Evidence that the penton base of adenovirus is involved in potentiation of toxicity of pseudomonas exotoxin conjugated to epidermal growth factor. Molecular Cell Biology 1984; 4:1528–1533

    CAS  Google Scholar 

  35. Wagner E, Plank C, Zatloukal K, Cotten M, Birnstiel ML: Influenza virus hemagglutinin HA-2 N-terminal fusogenic peptides augment gene transfer by transferrrin-polylysine-DNA complexes: toward a synthetic virus-like gene-transfer vehicle. Proc Natl Acad Sci USA 1992; 89:7934–7938

    Article  PubMed  CAS  Google Scholar 

  36. Morishita R, Gibbons GH, Ellison KE et al.: Single intraluminal delivery of antisense cdc2 kinase and proliferating-cell nuclear antigen oligonucleotides results in chronic inhibition of neointimal hyperplasia. Proc Natl Acad Sci USA 1993; 90

    Google Scholar 

  37. Pastore C, Feldman LJ, Perricaudet M, Steg PG: Intraluminal delivery of a pluronic gel enhances adenovirus-mediated arterial gene transfer: a morphometric study. Circulation 1994; 90:1–517

    Article  Google Scholar 

  38. March KL, Madison JE, Trapnell BC: Pharmacokinetics of adenoviral vector-mediated gene delivery to vascular smooth muscle cells: modulation by poloxamer 407 and implications for cardiovascular gene therapy. Human Gene Therapy 1995; 6:41–53

    Article  PubMed  CAS  Google Scholar 

  39. Takeshita S, Philbrook M, Rossow ST, Kim EA, Roth L: Successful gene transfer using photopolymerized hydrogel coatings applied onto endoluminal surface of the artery. Circulation 1994; 90:1–19

    Article  Google Scholar 

  40. Langer R: New methods of drug delivery. Science 1990; 249:1527–1533

    Article  PubMed  CAS  Google Scholar 

  41. Davis SS, Ilium L, Moghimi SM et al.: Microspheres for targeting drugs to specific body sites. J Controlled Release 1993; 24:157–163

    Article  CAS  Google Scholar 

  42. Cortesi R, Esposito E, Menegatti E, Gambari R, Nastruzzi C: Gelatin microspheres as a new approach for the controlled delivery of synthetic oligonucleotides and PCR-generated DNA fragments. Intl J Pharmaceutics 1994; 105:181–186.

    Article  CAS  Google Scholar 

  43. Sanderson JA, Kunz LL, Schroff RW et al.: Antibody-coated microspheres for drug delivery to prevent restenosis. Circulation 1994; 90:1–508

    Article  Google Scholar 

  44. Fritzberg AR, Vanderheyden J-L, Morgan AC, Schroff RW, Abrams PG: Rhenium-186/-188 labeled antibodies for radioimmunotherapy. Technetium and Rhenium in Chemistry and Nuclear Medicine 3, Cortina International — Verona 1990, pp 615–621

    Google Scholar 

  45. Schlom J: Basic principles and applications of monoclonal antibodies in the management of carcinoma: the Richard and Hinda Rosenthal Foundation award lecture. Cancer Research 1986; 46:3225–3238

    PubMed  CAS  Google Scholar 

  46. Ferkol T, Davis P, Kaetzel C et al.: Targeted gene delivery to respiratory epithelial cells. Pediatr Pulmonol Suppl. 1992; 8:236

    Google Scholar 

  47. Hashimoto Y, Sugawara M, Masuko T, Hojo H: Antitumor effect of actinomycinD entrapped in liposomes bearing subunhs of tumor-specific monoclonal immunoglobulin M antibody. Cancer Research 1983; 43:5328–5334

    PubMed  CAS  Google Scholar 

  48. Leserman LD, Barbet J, Kourilsky F, Weinstein JN: Targeting of cells to fluorescent liposomes covalently coupled with monoclonal antibody or protein A. Nature 1980; 288:602–604

    Article  PubMed  CAS  Google Scholar 

  49. Woodhouse CS, Bordonaro JP, Beaumier PL, Morgan AC: Second generation monoclonal antibodies to a human melanoma-associated proteoglycan. Human Melanoma Springer-Verlag — Berlin, 1990; pp 151–163, 1990.

    Google Scholar 

  50. Eary JF, Schroff RW, Abrams PG et al.: Successful imaging of malignant melanoma with technetium-99m-labeled monoclonal antibodies. J Nucl Medicine 1989; 30:25–32

    CAS  Google Scholar 

  51. Weiden PL, Breitz HB, Seiler CA et al.: Rhenium-186-labeled chimeric antibody NR-LU-13: pharmacokinetics, biodistribution and immunogenicity relative to murine analog NR-LU-10. J Nucl Medicine 1993; 34:2111–2119

    CAS  Google Scholar 

  52. Breitz HB, Weiden PL, Vanderheyden J-L et al.: Clinical experience with rhenium-186-labeled monoclonal antibodies for radioimmunotherapy: results of phase I trials. J Nucl Medicine 1992; 33:1099–1112

    CAS  Google Scholar 

  53. Gref R, Minamitake Y, Peracchia MT, Trubetskoy V, Torchilin V, Langer R: Biodegradable longcirculating polymeric nanospheres. Science 1994; 263:1600–1603

    Article  PubMed  CAS  Google Scholar 

Download references

Author information

Authors and Affiliations

  1. NeoRx Corporation, Seattle, Washington, USA

    Robert W. Schroff & Lawrence L. Kunz

Authors
  1. Robert W. Schroff
    View author publications

    You can also search for this author in PubMed Google Scholar

  2. Lawrence L. Kunz
    View author publications

    You can also search for this author in PubMed Google Scholar

Editor information

Editors and Affiliations

  1. Krannert Institute of Cardiology, Indiana University Medical Center, Indianapolis, Indiana, USA

    Keith L. March M.D., Ph.D.

  2. R. L. Roudebush VA Medical Center, Indianapolis, Indiana, USA

    Keith L. March M.D., Ph.D.

Rights and permissions

Reprints and permissions

Copyright information

© 1997 Springer Science+Business Media New York

About this chapter

Cite this chapter

Schroff, R.W., Kunz, L.L. (1997). Targeted and Sustained-Release Delivery Concepts in Gene Therapy. In: March, K.L. (eds) Gene Transfer in the Cardiovascular System. Developments in Cardiovascular Medicine, vol 189. Springer, Boston, MA. https://doi.org/10.1007/978-1-4615-6277-1_9

Download citation

  • DOI: https://doi.org/10.1007/978-1-4615-6277-1_9

  • Publisher Name: Springer, Boston, MA

  • Print ISBN: 978-1-4613-7881-5

  • Online ISBN: 978-1-4615-6277-1

  • eBook Packages: Springer Book Archive

Publish with us

Policies and ethics

Search

Navigation