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Lecture 22 Spring 2006 1 DNA delivery and DNA Vaccines ANNOUNCEMENTS: Last Time: intracellular drug delivery: enhancing cross priming for vaccines Today: DNA vaccination Reading: D.W. Pack, A.S. Hoffman, S. Pun, and P.S. Stayton, ‘Design and development of polymers for gene delivery,’ Nat. Rev. Drug Discov. 4 581-593 (2005) Supplementary Reading:

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Page 1: ANNOUNCEMENTS - MIT OpenCourseWare · Adv Drug Deliv Rev 30, 85-95 (1998). 7. Boussif, O. et al. A versatile vector for gene and oligonucleotide transfer into cells in culture and

Lecture 22 Spring 2006 1

DNA delivery and DNA Vaccines

ANNOUNCEMENTS:

Last Time: intracellular drug delivery: enhancing cross priming for vaccines

Today: DNA vaccination

Reading: D.W. Pack, A.S. Hoffman, S. Pun, and P.S. Stayton, ‘Design and development of polymers for gene delivery,’ Nat. Rev. DrugDiscov. 4 581-593 (2005)

Supplementary Reading:

Page 2: ANNOUNCEMENTS - MIT OpenCourseWare · Adv Drug Deliv Rev 30, 85-95 (1998). 7. Boussif, O. et al. A versatile vector for gene and oligonucleotide transfer into cells in culture and

Lecture 22 Spring 2006 2

Models of membrane-penetrating peptide function

Penetratin:Short peptide sequence from drosophila transcription factor protein Antennapedia

RQIKIWFQNRRMKWKK

DIRECT ENTRY TO CYTOSOL:MEMBRANE-PENETRATING PEPTIDESDIRECT ENTRY TO CYTOSOL:MEMBRANE-PENETRATING PEPTIDES

DIRECT ENTRY TO CYTOSOL:MEMBRANE-PENETRATING PEPTIDES

Image removed due to copyright restrictions.Please see: Derossi, et al. J Biol Chem 271, no. 30 (1996): 18188.

Page 3: ANNOUNCEMENTS - MIT OpenCourseWare · Adv Drug Deliv Rev 30, 85-95 (1998). 7. Boussif, O. et al. A versatile vector for gene and oligonucleotide transfer into cells in culture and

Lecture 22 Spring 2006 3

ACTIVATION ON ENTRY TO THE CYTOSOLSelective bond dissociation using reversible disulfide linkages

Image removed due to copyright restrictions.Please see: Falnes. Curr Opin Cell Biol 12 (2000): 407.

Page 4: ANNOUNCEMENTS - MIT OpenCourseWare · Adv Drug Deliv Rev 30, 85-95 (1998). 7. Boussif, O. et al. A versatile vector for gene and oligonucleotide transfer into cells in culture and

Lecture 22 Spring 2006 4

Pore-forming proteins/peptides as a tool for membrane-penetrating drug carriers

DIRECT ENTRY TO CYTOSOL:PORE-FORMING PEPTIDESDIRECT ENTRY TO CYTOSOL:PORE-FORMING PEPTIDES

DIRECT ENTRY TO CYTOSOL:PORE-FORMING PEPTIDES

Figures 1A, 1B, and 1C removed due to copyright restrictions.Please see: Bhakdi. Arch Microbiol 165 (1996): 73.

Page 5: ANNOUNCEMENTS - MIT OpenCourseWare · Adv Drug Deliv Rev 30, 85-95 (1998). 7. Boussif, O. et al. A versatile vector for gene and oligonucleotide transfer into cells in culture and

Lecture 22 Spring 2006 5

fusogenic peptides: using viral entry strategies for drug deliveryDIRECT ENTRY TO CYTOSOL:

FUSOGENIC PEPTIDESDIRECT ENTRY TO CYTOSOL:FUSOGENIC PEPTIDES

DIRECT ENTRY TO CYTOSOL:FUSOGENIC PEPTIDES

Image removed due to copyright restrictions.Please see: Hawiger. Curr Opin Chem Biol 3 (1999): 89.

Page 6: ANNOUNCEMENTS - MIT OpenCourseWare · Adv Drug Deliv Rev 30, 85-95 (1998). 7. Boussif, O. et al. A versatile vector for gene and oligonucleotide transfer into cells in culture and

Lecture 22 Spring 2006 6

DNA DELIVERY AND DNA VACCINES

Page 7: ANNOUNCEMENTS - MIT OpenCourseWare · Adv Drug Deliv Rev 30, 85-95 (1998). 7. Boussif, O. et al. A versatile vector for gene and oligonucleotide transfer into cells in culture and

Lecture 22 Spring 2006 7

GENE THERAPY FOR VACCINATION: GENES THAT ENCODE ANTIGEN

Dendritic cell

CD4+ T cells

CD8+ T cellsTissue stromal cell

Page 8: ANNOUNCEMENTS - MIT OpenCourseWare · Adv Drug Deliv Rev 30, 85-95 (1998). 7. Boussif, O. et al. A versatile vector for gene and oligonucleotide transfer into cells in culture and

Lecture 22 Spring 2006 8

Motivation for DNA vaccinesWhy are synthetic vectors of interest?

Page 9: ANNOUNCEMENTS - MIT OpenCourseWare · Adv Drug Deliv Rev 30, 85-95 (1998). 7. Boussif, O. et al. A versatile vector for gene and oligonucleotide transfer into cells in culture and

Lecture 22 Spring 2006 9

idealized objectives of DNA delivery

2 classes of synthetic vectors we’ll discuss:

Image removed due to copyright restrictions.Please see: Vijayanathan, et al. Biochemistry 41, no. 48 (2002): 10485.

Page 10: ANNOUNCEMENTS - MIT OpenCourseWare · Adv Drug Deliv Rev 30, 85-95 (1998). 7. Boussif, O. et al. A versatile vector for gene and oligonucleotide transfer into cells in culture and

Lecture 22 Spring 2006 10

Image removed due to copyright restrictions.Please see: Segura, T., and L. D. Shea. “Materials for non-viral gene delivery.”Annual Review of Materials Research 31 (2001): 25-46.

Page 11: ANNOUNCEMENTS - MIT OpenCourseWare · Adv Drug Deliv Rev 30, 85-95 (1998). 7. Boussif, O. et al. A versatile vector for gene and oligonucleotide transfer into cells in culture and

Lecture 22 Spring 2006 11

Polyplex formation between polycations and plasmid DNA

Image removed due to copyright restrictions.Please see: Pack, et al. Nat Rev Drug Discov (2005).

Page 12: ANNOUNCEMENTS - MIT OpenCourseWare · Adv Drug Deliv Rev 30, 85-95 (1998). 7. Boussif, O. et al. A versatile vector for gene and oligonucleotide transfer into cells in culture and

Lecture 22 Spring 2006 12

Packaging DNA for delivery and cytosolic release

Image removed due to copyright restrictions. Please see: Oupicky, D., A. L. Parker, and L.W. Seymour. “Laterally stabilized Complexes of DNA with Linear Reducible Polycations: Strategy for Triggered Intracellular Activation ofDNA Delivery Vectors.” J Am Chem Soc 124 (2002): 8-9.

Page 13: ANNOUNCEMENTS - MIT OpenCourseWare · Adv Drug Deliv Rev 30, 85-95 (1998). 7. Boussif, O. et al. A versatile vector for gene and oligonucleotide transfer into cells in culture and

Lecture 22 Spring 2006 13

Polycation/DNA charge ratios in DNA packaging and release

Image removed due to copyright restrictions.Please see: Segura et al. Biomaterials 26 (2005): 1575-1584.

Page 14: ANNOUNCEMENTS - MIT OpenCourseWare · Adv Drug Deliv Rev 30, 85-95 (1998). 7. Boussif, O. et al. A versatile vector for gene and oligonucleotide transfer into cells in culture and

Lecture 22 Spring 2006 14

Substrate-mediated DNA delivery

Segura et al. Biomaterials 26 1575-1584 (2005) NIH 3T3 fibroblasts

Page 15: ANNOUNCEMENTS - MIT OpenCourseWare · Adv Drug Deliv Rev 30, 85-95 (1998). 7. Boussif, O. et al. A versatile vector for gene and oligonucleotide transfer into cells in culture and

Lecture 22 Spring 2006 15

Lipid-DNA microstructures

Image removed due to copyright restrictions.Please see: http://avantipolarlipids.com/

http://avantipolarlipids.com/
Page 16: ANNOUNCEMENTS - MIT OpenCourseWare · Adv Drug Deliv Rev 30, 85-95 (1998). 7. Boussif, O. et al. A versatile vector for gene and oligonucleotide transfer into cells in culture and

Lecture 22 Spring 2006 16

Lipid-DNA microstructures

Figure removed due to copyright restrictions.Please see: Figure 1 in Koltover, et al. Science 281 (1998): 78-81.

Page 17: ANNOUNCEMENTS - MIT OpenCourseWare · Adv Drug Deliv Rev 30, 85-95 (1998). 7. Boussif, O. et al. A versatile vector for gene and oligonucleotide transfer into cells in culture and

Lecture 22 Spring 2006 17

Lipid-DNA microstructures

Image removed due to copyright restrictions. Please see: Martin-Herranz, A. et al. “Surface Functionalized Cationic Lipid-DNA Complexes for Gene Delivery: PEGylated Lamellar Complexes Exhibit Distinct DNA-DNA Interaction Regimes. Biophys J 86 (2004): 1160-8.

Page 18: ANNOUNCEMENTS - MIT OpenCourseWare · Adv Drug Deliv Rev 30, 85-95 (1998). 7. Boussif, O. et al. A versatile vector for gene and oligonucleotide transfer into cells in culture and

Lecture 22 Spring 2006 18

LIPID AND PARTICLE-BASED DNA CARRIERS

Image removed due to copyright restrictions.

Page 19: ANNOUNCEMENTS - MIT OpenCourseWare · Adv Drug Deliv Rev 30, 85-95 (1998). 7. Boussif, O. et al. A versatile vector for gene and oligonucleotide transfer into cells in culture and

Lecture 22 Spring 2006 19

TRANSPORT FROM THE CYTOSOL TO THE NUCLEUS

Figure removed due to copyright restrictions. Please see: Figure 1 in Escriou et al. Adv Drug Delib Rev 55 (2003): 295.

Page 20: ANNOUNCEMENTS - MIT OpenCourseWare · Adv Drug Deliv Rev 30, 85-95 (1998). 7. Boussif, O. et al. A versatile vector for gene and oligonucleotide transfer into cells in culture and

Lecture 22 Spring 2006 20

Figure removed due to copyright restrictions. Please see: Figure 3 in Kircheis et al. Adv Drug Delib Rev 53 (2001): 2341.

Page 21: ANNOUNCEMENTS - MIT OpenCourseWare · Adv Drug Deliv Rev 30, 85-95 (1998). 7. Boussif, O. et al. A versatile vector for gene and oligonucleotide transfer into cells in culture and

Lecture 22 Spring 2006 21

Limitations of current materials

Graph removed due to copyright restrictions.Please see: Moghimi , et al. Mol Therapy 11 (2005): 990-995.

Page 22: ANNOUNCEMENTS - MIT OpenCourseWare · Adv Drug Deliv Rev 30, 85-95 (1998). 7. Boussif, O. et al. A versatile vector for gene and oligonucleotide transfer into cells in culture and

Lecture 22 Spring 2006 22

Built-in adjuvants: DNA vaccines encoding antigen and other factors

Figure removed due to copyright restrictions. Please see: Figure 1 in Sumida, et al. J Clinical Invest 114 (2004): 1334-1342.

Page 23: ANNOUNCEMENTS - MIT OpenCourseWare · Adv Drug Deliv Rev 30, 85-95 (1998). 7. Boussif, O. et al. A versatile vector for gene and oligonucleotide transfer into cells in culture and

Lecture 22 Spring 2006 23

Built-in adjuvants: DNA vaccines encoding antigen and other factors

Graph removed due to copyright restrictions.Please see: Sumida et al. J. Clinical Invest 114 (2004): 1334-1342.

Page 24: ANNOUNCEMENTS - MIT OpenCourseWare · Adv Drug Deliv Rev 30, 85-95 (1998). 7. Boussif, O. et al. A versatile vector for gene and oligonucleotide transfer into cells in culture and

Lecture 22 Spring 2006 24

Engineering the function of antigen presenting cells by DNA delivery

Page 25: ANNOUNCEMENTS - MIT OpenCourseWare · Adv Drug Deliv Rev 30, 85-95 (1998). 7. Boussif, O. et al. A versatile vector for gene and oligonucleotide transfer into cells in culture and

Lecture 22 Spring 2006 25

Further Reading 1. Varga, C. M., Hong, K. & Lauffenburger, D. A. Quantitative analysis of synthetic gene delivery vector design

properties. Mol Ther 4, 438-46 (2001). 2. Varga, C. M., Wickham, T. J. & Lauffenburger, D. A. Receptor-mediated targeting of gene delivery vectors:

insights from molecular mechanisms for improved vehicle design. Biotechnol Bioeng 70, 593-605 (2000). 3. Segura, T. & Shea, L. D. Materials for non-viral gene delivery. Annual Review of Materials Research 31, 25-46

(2001). 4. Segura, T. & Shea, L. D. Surface-tethered DNA complexes for enhanced gene delivery. Bioconjugate Chemistry

13, 621-629 (2002). 5. Vijayanathan, V., Thomas, T. & Thomas, T. J. DNA nanoparticles and development of DNA delivery vehicles for

gene therapy. Biochemistry 41, 14085-94 (2002). 6. Demeneix, B. et al. Gene transfer with lipospermines and polyethylenimines. Adv Drug Deliv Rev 30, 85-95

(1998). 7. Boussif, O. et al. A versatile vector for gene and oligonucleotide transfer into cells in culture and in vivo:

polyethylenimine. Proc Natl Acad Sci U S A 92, 7297-301 (1995). 8. Zanta, M. A., Boussif, O., Adib, A. & Behr, J. P. In vitro gene delivery to hepatocytes with galactosylated

polyethylenimine. Bioconjug Chem 8, 839-44 (1997). 9. Rungsardthong, U. et al. Effect of polymer ionization on the interaction with DNA in nonviral gene delivery

systems. Biomacromolecules 4, 683-90 (2003). 10. Rungsardthong, U. et al. Copolymers of amine methacrylate with poly(ethylene glycol) as vectors for gene

therapy. J Control Release 73, 359-80 (2001). 11. Oupicky, D., Parker, A. L. & Seymour, L. W. Laterally stabilized complexes of DNA with linear reducible

polycations: strategy for triggered intracellular activation of DNA delivery vectors. J Am Chem Soc 124, 8-9 (2002).

12. Ewert, K. et al. Cationic lipid-DNA complexes for gene therapy: understanding the relationship between complex structure and gene delivery pathways at the molecular level. Curr Med Chem 11, 133-49 (2004).

13. Martin-Herranz, A. et al. Surface functionalized cationic lipid-DNA complexes for gene delivery: PEGylated lamellar complexes exhibit distinct DNA-DNA interaction regimes. Biophys J 86, 1160-8 (2004).

14. Bonifaz, L. C. et al. In Vivo Targeting of Antigens to Maturing Dendritic Cells via the DEC-205 Receptor Improves T Cell Vaccination. J Exp Med 199, 815-24 (2004).

15. Kircheis, R., Wightman, L. & Wagner, E. Design and gene delivery activity of modified polyethylenimines. Advanced Drug Delivery Reviews 53, 341-358 (2001).


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