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Non viral methods that have been developed for DNA delivery

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It gives opportunity for treating diseases resulting from single gene ... and self disassembly with DNA. Gene delivery vectors : ADVANTAGES and DISADVANTAGES ... – PowerPoint PPT presentation

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Title: Non viral methods that have been developed for DNA delivery


1
Non viral methods that have been developed for
DNA delivery
2
What makes Gene Therapy so attractive nowadays ?
  • It gives opportunity for treating diseases
    resulting from single gene to complex gene
    disorders such as CARDIOVASCULAR DISEASE,
    DIABETES, HYPERTENSION, and MALIGNANCY.

How ?
  • By making the cell produce a therapeutic protein

3
Review of the main steps involved in gene
transfer
  • Delivery of DNA to the target site
  • Cellular penetration and internalization
  • Nuclear translocation
  • Transcription and translation
  • Protein processing

4
Gene delivery barriers
5
Methods for Introducing DNA into the cell
  • Specified according to the type of the Gene
    Delivery Vector
  • Recombinant Retrovirus and Adenovirus Mediated
    DNA Delivery ?Viral vectors

Adenovirus/PEI/DNA complex
Baker at al. Gene Ther. 4 1997
6
  • Liposome and Polymer Mediated DNA delivery
  • ? Non viral vectors
  • LIPOSOMES

7
DNA cationic liposome complexes
8
  • Cationic Polymers

9
Charge Shifting Cationic Polymers promoting
self assembly and self disassembly with DNA

10
Gene delivery vectors ADVANTAGES and
DISADVANTAGES
  • VIRUSES
  • Very efficient, but
  • Can be immunogenic Not good for repeat dose,
    body tends to reject them
  • Can be potentially toxic
  • Can activate oncogenes
  • Difficult to prepare

11
  • LIPOSOMES
  • Not very efficient
  • Can be toxic
  • POLYMERS
  • Their properties can be easily controlled, so
    that they can be designed to be
  • biocompatible - non-toxic and non-immunogenic
  • biodegradable
  • and more efficient.

12
Conclusions
  • Many of the barriers limiting the success of
    Gene Therapy have been discovered and alternative
    solutions have been given.
  • Significant improvement of the transfection
    efficiency was achieved by testing different
    techniques.
  • The polymer materials have turned out to be one
    of the promising strategy for gene delivery
    giving a big variety of opportunities for
    modeling of one more flexible system.

13
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