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    Gene Therapy: Khadija Tariq Bs-Genetics

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    khadija tariq
    This document discusses gene therapy, including its history, types, vectors used, and applications. It defines gene therapy as using genetic material to cure or prevent diseases, which can be done by replacing, inactivating, or introducing new genes. The two main types are somatic gene therapy, which affects only non-reproductive cells and has non-heritable effects but requires repeated treatment, and germline gene therapy, which affects heritable germ line cells but has unknown long-term side effects. Viral and non-viral vectors are used to deliver genetic material to host cells, with viruses commonly used but also having safety risks. Gene therapy has potential applications in genetic engineering and gene doping.

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    Gene Therapy: Khadija Tariq Bs-Genetics

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    khadija tariq
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    This document discusses gene therapy, including its history, types, vectors used, and applications. It defines gene therapy as using genetic material to cure or prevent diseases, which can be done by replacing, inactivating, or introducing new genes. The two main types are somatic gene therapy, which affects only non-reproductive cells and has non-heritable effects but requires repeated treatment, and germline gene therapy, which affects heritable germ line cells but has unknown long-term side effects. Viral and non-viral vectors are used to deliver genetic material to host cells, with viruses commonly used but also having safety risks. Gene therapy has potential applications in genetic engineering and gene doping.

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    This document discusses gene therapy, including its history, types, vectors used, and applications. It defines gene therapy as using genetic material to cure or prevent diseases, which can be done by replacing, inactivating, or introducing new genes. The two main types are somatic gene therapy, which affects only non-reproductive cells and has non-heritable effects but requires repeated treatment, and germline gene therapy, which affects heritable germ line cells but has unknown long-term side effects. Viral and non-viral vectors are used to deliver genetic material to host cells, with viruses commonly used but also having safety risks. Gene therapy has potential applications in genetic engineering and gene doping.

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    © All Rights Reserved
    Download as PPTX, PDF, TXT or read online from Scribd
    Download as pptx, pdf, or txt
    36 views6 pages

    Gene Therapy: Khadija Tariq Bs-Genetics

    Uploaded by

    khadija tariq
    This document discusses gene therapy, including its history, types, vectors used, and applications. It defines gene therapy as using genetic material to cure or prevent diseases, which can be done by replacing, inactivating, or introducing new genes. The two main types are somatic gene therapy, which affects only non-reproductive cells and has non-heritable effects but requires repeated treatment, and germline gene therapy, which affects heritable germ line cells but has unknown long-term side effects. Viral and non-viral vectors are used to deliver genetic material to host cells, with viruses commonly used but also having safety risks. Gene therapy has potential applications in genetic engineering and gene doping.

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    of 6

    GENE THERAPY

    Khadija Tariq
    Bs-Genetics
    What is gene therapy?
    • Use of genetic material to cure or prevent diseases.
    • Can be done in different ways through different methods
     Replacing genes
     Inactivating genes
     Introducing new genes

    • Clinical trials and ethical boundaries


    History of gene therapy
    • 1960s experiments on HPRT deficient cell,
    confirming gene transfer
    • Late 1960s experiments on papovaviruses SV40
    and plyoma
    • 1970s recombinant DNA technology
    • 1979 clinical trials of Marin Cline
    • 1981-2 use of retroviral vectors with 100% efficiency
    • Banbury conference in 1983 discussing techniques
    on gene therapy
    • 1989-90 human clinical trial approved
    Types of gene therapy
    Somatic gene therapy Germ line gene therapy
    • The methods in which • The methods in which the
    somatic or non-reproductive germ line cells are
    cells are transformed transformed
    • Types
     In-vivo - the methods in which the cells are
    • The affects are inheritable
    transformed inside the body to the next generation
     Ex-vivo – the methods in which the cells are
    transformed outside the body • The side effects are
    • The affects are non- unknown
    heritable • Banned in a lot of
    • Drawbacks include short countries of the world.
    lived affects/ repeated
    therapy
    Vectors
    Viral vectors Non-viral vectors

    • The genetic material is • The genetic material is


    targeted to host cells via targeted through non-viral
    genetically engineered methods such as
    viruses
    • Naked DNA
    • Viruses used are retroviruses,
    Such as plasmid injections
    adenoviruses etc.
    • Oligonucleotides
    • Depends on the infection,
    Bind to transcription factors to
    host spectrum and transgene
    prevent expression of a faulty gene
    expression of the virus
    • Lipoplexes
    • Drawbacks include
    Liposomal structures to protect DNA
    overexpression, wide host
    spectrum etc.
    Applications

    • Gene doping

    • Genetic engineering

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