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    8.8 What Is Gene Therapy

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    Narasimha Murthy

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    © All Rights Reserved
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    8.8 What Is Gene Therapy

    Uploaded by

    Narasimha Murthy
    17 views2 pages

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    8.8 What is Gene Therapy

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    17 views2 pages

    8.8 What Is Gene Therapy

    Uploaded by

    Narasimha Murthy

    Copyright:

    © All Rights Reserved
    Download as DOCX, PDF, TXT or read online from Scribd
    Download as docx, pdf, or txt
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    What is Gene therapy?

    Gene therapy is a technique which involves the replacement of defective genes with healthy
    ones in order to treat genetic disorders. It is an artificial method that introduces DNA into
    the cells of the human body. The first gene therapy was successfully accomplished in the
    year 1989.

    The simple process of gene therapy is shown in the figure below:

    In the figure, the cell with the defective gene is injected with a normal gene which helps in
    the normal functioning of the cell. This technique is employed mainly to fight against the
    diseases in the human body and also to treat genetic disorders. The damaged proteins are
    replaced in the cell by the insertion of DNA into that cell. Generally, improper protein
    production in the cell leads to diseases. These diseases are treated using a gene therapy
    technique. For example, cancer cells contain faulty cells which are different from the normal
    cells and have defective proteins. Hence, if these proteins are not replaced, this disease
    would prove to be fatal.

    Types of Gene Therapy

    Basically, there are two types of gene therapy

     Somatic Gene Therapy

    This type usually occurs in the somatic cells of human body. This is related to a single
    person and the only person who has the damaged cells will be replaced with healthy cells. In
    this method, therapeutic genes are transferred into the somatic cells or the stem cells of the
    human body. This technique is considered as the best and safest method of gene therapy.

     Germline Gene Therapy

    It occurs in the germline cells of the human body. Generally, this method is adopted to treat
    the genetic, disease causing-variations of genes which are passed from the parents to their
    children. The process involves introducing a healthy DNA into the cells responsible for
    producing reproductive cells, eggs or sperms. Germline gene therapy is not legal in many
    places as the risks outweigh the rewards.

    Application of Gene Therapy

    1. It is used in the replacement of genes that cause medical ill-health


    2. The method generally destroys the problem causing genes
    3. It helps the body to fight against diseases by adding genes to the human body
    4. This method is employed to treat diseases such as cancer, ADA deficiency, cystic
    fibrosis, etc.

    The first clinical gene therapy was given in 1990 to a 4 - year old girl with adenosine
    deaminase (ADA) deficiency. This enzyme is very important for the immune system to
    function. SCID is caused due to defect in the gene for the enzyme adenosine deaminase.

    Gene therapy holds promise for treating a wide range of diseases, such as cancer, cystic
    fibrosis, heart disease, diabetes, hemophilia and AIDS.

    TRANSFER OF GENETIC MATERIAL

    1. Transfection is the process of deliberately introducing naked or purified nucleic


    acids into eukaryotic cells.

    2. "Transformation" is typically used to describe non-viral DNA transfer in bacteria


    and non-animal eukaryotic cells, including plant cells. In animal cells, transfection is
    the preferred term as transformation is also used to refer to progression to a
    cancerous state (carcinogenesis) in these cells.

    3. Transduction is often used to describe virus-mediated gene transfer into eukaryotic


    cells.

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