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    Human Gene Therapy

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    Anupriya Singh
    This document discusses human gene therapy. It describes how gene therapy works by introducing genes into cells to cure or prevent disease. There are two main types of gene therapy: somatic cell gene therapy, which treats only the individual, and germline gene therapy, which could be inherited. Viruses and non-viral methods can be used as vectors to deliver therapeutic genes. While gene therapy has potential benefits, there are also safety risks to address.

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    Human Gene Therapy

    Uploaded by

    Anupriya Singh
    70 views18 pages
    This document discusses human gene therapy. It describes how gene therapy works by introducing genes into cells to cure or prevent disease. There are two main types of gene therapy: somatic cell gene therapy, which treats only the individual, and germline gene therapy, which could be inherited. Viruses and non-viral methods can be used as vectors to deliver therapeutic genes. While gene therapy has potential benefits, there are also safety risks to address.

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    This document discusses human gene therapy. It describes how gene therapy works by introducing genes into cells to cure or prevent disease. There are two main types of gene therapy: somatic cell gene therapy, which treats only the individual, and germline gene therapy, which could be inherited. Viruses and non-viral methods can be used as vectors to deliver therapeutic genes. While gene therapy has potential benefits, there are also safety risks to address.

    Copyright:

    © All Rights Reserved
    Download as PPTX, PDF, TXT or read online from Scribd
    Download as pptx, pdf, or txt
    70 views18 pages

    Human Gene Therapy

    Uploaded by

    Anupriya Singh
    This document discusses human gene therapy. It describes how gene therapy works by introducing genes into cells to cure or prevent disease. There are two main types of gene therapy: somatic cell gene therapy, which treats only the individual, and germline gene therapy, which could be inherited. Viruses and non-viral methods can be used as vectors to deliver therapeutic genes. While gene therapy has potential benefits, there are also safety risks to address.

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    © All Rights Reserved
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    of 18

    HUMAN GENE

    THERAPY

    SUBMITTED TO SUBMITTED BY
    Dr. SHIPRA SHUKLA DOLLY
    MSC (BIOTECH.)
    3rd SEM.2018-20
    INTRODUCTION
    • Gene therapy is the introduction of genes in to
    existing cells to prevent or cure a wide range
    of disease.
    • It is a technique for correcting defective genes
    responsible for disease development .
    • The first approved gene therapy experiment
    occurred on september 14,1990 in US, when
    Ashanti De Silva was treated for ADA-SCID.
    TYPES OF GENE THERAPY
    SOMATIC CELL GENE THERAPY GERM LINE GENE THERAPY
    • Therapeutic genes • Therapeutic genes
    transferred into the somatic transferred in to the germ
    cell cell.
    • Eg.Introduction of genes • Eg. Genes introduced into
    into bone marrow cells eggs and sperms.
    ,blood cells,skin cells, etc. • It is heritable and passed on
    • Will not be inherited later to later generations .
    generations. • For safety ,ethical and
    • At present all researches technical reasons , it is not
    directed to correct genetic being attempted at present.
    defects in cells.
    EXAMPLE OF GENE THERAPY
    • 1st gene therapy-to correct deficiency of
    enzyme, Adenosine deaminase (ADA).
    • Performed on a 4yr. Old girl Ashanti De Silva.
    • Was suffering from SCID-severe combined
    immunodeficiency.
    • Caused due to defect in gene coding for ADA.
    IN VIVO GENE THERAPY
    • Direct delivery of thereputic gene into target
    cell into patients body.
    • Carried out by viral or non viral vector system.
    • In vivo gene transfer is necessary when culture
    cells cannot be re –implanted in patients
    effectively.
    EXAMPLE OF IN VIVO GENE THERAPY
    • In patients with cystic fibrosis ,a protein called
    cystic fibrosis transmembrane regulator
    (CFTR) is absent due to a defect.
    • In the absence of CFTR chloride ions
    concentrate with in the cells and it draws
    water from surrounding.
    • Treated by in vivo replacement of defective
    gene by adenovirus vector.
    VECTOR IN GENE THERAPY
    • To transfer the desired gene into a target cell,a
    carrier is required such vehicles of gene
    delivery are known as vectors.
    • 2 main classes
    Viral vectors
    Non viral vectors
    VIRAL VECTORS
    1. RETROVIRUS VECTOR SYSTEM
     Can carry a DNA of size-less than 3.4kb.
     Target cell –dividing
    2. ADENOVIRUS VECTOR SYSTEM
     Adeno virus with a DNA genome-good vectors.
     Target-non dividing human cell.
     Eg. Common cold , adenovirus.
    3. ADENO ASSOCIATED VIRUS VECTOR
    4. HERPEX SIMPLEX VIRUS VECTOR
    NON VIRAL VECTOR SYSTEM
    1. PURE DNA CONSTRUCT
    2. LIPOPLEXES
    3. DNA MOLECULAR CONJUGATES
    4. HUMAN ARTIFICIAL CHROMOSOMES
    METHODS OF GENE DELIVERY
    PHYSICAL METHOD
    GENE GUN
    MICRO INJECTION
    MICRO MANIPULATIOR
    CHEMICAL METHOD
    USING DETERGENT MITURES
    LIPOFECTION
    OTHER TYPES OF GENE THERAPY
    GENE AUGMENTATION THERAPY
    Most common form of gene therapy
    Eg. Replacement of defective p53 gene by a
    normal one in liver cancer.
    GENE INHIBITION THERAPY
     Two types- antigen and antisense therapy
     Antigen
     Antisense
    ADVANTAGES
    o Gene therapy has the potential to eliminate
    and prevent hereditary disease such as cystic
    fibrosis ,ADA-SCID etc.
    o It is a possible cure for heart disease, AIDS and
    cancer .
    o It is gives someone born with a genetic
    disease a chance to live.
    o It can be used to eradicate disease from the
    future generations.
    DISADVANTAGES
    o Immune response to the transferred gene
    stimulates a potential risk to gene therapy .
    o Viruses used as vector for gene transfer may
    cause toxicity ,immune responses, and
    inflammatory reaction in the host .
    o Disorders caused by defects in multiple genes
    can not be treated effectively using gene
    therapy

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