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    Kaan Uğur
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    Gene therapy is a potential treatment approach for genetic disorders that are resistant to traditional therapies. It involves introducing new genetic instructions into patient tissues to compensate for abnormal or missing genes. While many success stories have emerged in recent years for diseases like ADA-SCID, viral vectors like adenovirus are commonly used but can elicit strong immune responses and hepatotoxicity when administered systemically. Non-viral vectors may provide an alternative but typically have lower transfection efficiency than viruses.

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    Lecture 5 (1)

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    Gene therapy is a potential treatment approach for genetic disorders that are resistant to traditional therapies. It involves introducing new genetic instructions into patient tissues to compensate for abnormal or missing genes. While many success stories have emerged in recent years for diseases like ADA-SCID, viral vectors like adenovirus are commonly used but can elicit strong immune responses and hepatotoxicity when administered systemically. Non-viral vectors may provide an alternative but typically have lower transfection efficiency than viruses.

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    6 views4 pages

    Lecture 5

    Uploaded by

    Kaan Uğur
    Gene therapy is a potential treatment approach for genetic disorders that are resistant to traditional therapies. It involves introducing new genetic instructions into patient tissues to compensate for abnormal or missing genes. While many success stories have emerged in recent years for diseases like ADA-SCID, viral vectors like adenovirus are commonly used but can elicit strong immune responses and hepatotoxicity when administered systemically. Non-viral vectors may provide an alternative but typically have lower transfection efficiency than viruses.

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    © All Rights Reserved
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    of 4

    Gene Therapy

    Gene Therapy
    - has emerged in the late 20th century

    - a potential approach to the treatment of


    genetic disorders which are resistant to
    traditional therapies
    - to cure genetic diseases by introducing new
    genetic instructions into the tissues of patients
    in order to compensate for abnormal or missing
    genes or to convey a new function

    - cancer, cardiovascular, infectious, eye or


    lung diseases
    - Many success stories in the recent years (ALD,
    Example: Hemophilia A gene
    inherited retinal diseases, SCID) therapy
    http://www.nwabr.org/studentbiotech/winners/studentwork/
    2007/WB_BA_TRONGTHAM/1_index_2.htm
    Gene Therapy - vectors
    - Viral vs non-viral vectors

    - Viral:
     Ad
     AAV
     Lentivirus
     Retrovirus
     SFV

    - Non-viral vectors:
     Liposomes
     Polymer-based nanoparticles
     Dendrimers
    http://www.biochem.arizona.edu/classes/bioc471/
    pages/Lecture25/Lecture25.html
    Adenovirus (Ad)
    - the most commonly used knob

    viral vector in gene therapy shaft


    fiber
    studies
    - high transfection efficiency
    in a wide variety of cells
    - can be amplified at very
    high titers.
    RGD
    motif
     Systemic administration penton
    • Elicit strong immune responses,
    hepatotoxicity
    • Show decreased blood circulation pXI

    hexon

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