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  • Gene Therapy One

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    Aminath Meesan
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    Gene therapy is the use of DNA as a pharmaceutical to treat disease by supplementing or altering genes within cells. It commonly replaces a mutated gene with functional DNA encoding a therapeutic gene, but can also directly correct mutations or encode therapeutic proteins instead of natural human genes. DNA encoding a therapeutic protein is packaged in a vector to transport it into cells, where it is expressed to produce the protein and treat the patient's disease. Gene therapy was first proposed in 1972 and the first FDA-approved human trial occurred in 1990 for ADA-SCID.

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    basic ideas on gene therapy

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    Gene therapy is the use of DNA as a pharmaceutical to treat disease by supplementing or altering genes within cells. It commonly replaces a mutated gene with functional DNA encoding a therapeutic gene, but can also directly correct mutations or encode therapeutic proteins instead of natural human genes. DNA encoding a therapeutic protein is packaged in a vector to transport it into cells, where it is expressed to produce the protein and treat the patient's disease. Gene therapy was first proposed in 1972 and the first FDA-approved human trial occurred in 1990 for ADA-SCID.

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    3 views1 page

    Gene Therapy One

    Uploaded by

    Aminath Meesan
    Gene therapy is the use of DNA as a pharmaceutical to treat disease by supplementing or altering genes within cells. It commonly replaces a mutated gene with functional DNA encoding a therapeutic gene, but can also directly correct mutations or encode therapeutic proteins instead of natural human genes. DNA encoding a therapeutic protein is packaged in a vector to transport it into cells, where it is expressed to produce the protein and treat the patient's disease. Gene therapy was first proposed in 1972 and the first FDA-approved human trial occurred in 1990 for ADA-SCID.

    Copyright:

    Attribution Non-Commercial (BY-NC)
    Download as DOCX, PDF, TXT or read online from Scribd
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    of 1

    Gene therapy

    Gene therapy is the use ofDNA as a pharmaceutical agent to treat disease. It derives its name from the idea that DNA can be used to supplement or alter genes within an individual's cells as a therapy to treat disease. The most common form of gene therapy involves using DNA that encodes a functional, therapeutic gene to replace a mutated gene. Other forms involve directly correcting a mutation, or using DNA that encodes a therapeutic protein drug (rather than a natural human gene) to provide treatment. In gene therapy, DNA that encodes a therapeutic protein is packaged within a "vector", which is used to get the DNA inside cells within the body. Once inside, the DNA becomes expressed by the cell machinery, resulting in the production of therapeutic protein, which in turn treats the patient's disease. Gene therapy was first conceptualized in 1972, with the authors urging caution before commencing gene therapy studies in humans. The first FDA-approved gene therapy experiment in the United States occurred in 1990, when Ashanti DeSilva was treated forADA-SCID

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