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Module 1: Gene Delivery and Other Variants

    Study Reminders
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    Carriers are needed for putting genes inside cells: Viruses, Polymers or Lipids.

    Viral Vectors

    Advantages
    High transfection efficiency
    Natural tropism
    Evolved mechanisms for endosomal escape
    Natural transportation mechanism of DNA into the nucleus
    Disadvantages
    The strong immune reaction against viral proteins prohibit multiple administrations
    Possibility of chromosomal insertion and proto-oncogene activation
    Complicated synthesis process
    Limitation on gene size
    Toxicity, contamination of the live virus
    Non-Viral Vectors
    Advantages
    Low immunogenicity
    Can be made to be non-toxic
    Easy to synthesis: quality control for mass production
    Potentially targetable
    No limit on plasmid size
    No integration: can be administered as drugs
    Disadvantages
    Low transfection efficacy
    Currently, most vehicles are toxic at high dosages
    No natural tropism, endosomal escape or nuclear transport mechanisms
    How are genes used as vaccines?
    DNA vaccines: Genes encoded for specific antigenic proteins of interest. Instead of delivering the pathogen or recombinant protein, the host cells themselves produce the antigen

    Immunization against allergies:
    - Sensitization
    - Re-exposure
    - IgE mediated anaphylaxis
    - Immunize
    - IgG2a mediated Th1 response
    - Allergen Exposure

    Gene Carriers:
    - Polyethyleneimine
    - Chitosan
    - PAMAM-dendrimers
    - Novel cationic polymers

    Cancer Vaccines:
    - Prophylactic vaccine- prevent liver and cervical cancers
    - Therapeutic vaccine- increase the immune response against tumour antigens