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About The Gene Therapy


Gene therapy is an experimental technique that uses genes to treat or prevent disease. The s explain what is gene tharapy? Types of gene therapy. – PowerPoint PPT presentation

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Title: About The Gene Therapy

Gene Therapy
History of Gene Therapy
Gene therapy is the use of genetic material- DNA
or RNA- as a medicine. Classical gene therapy
involves the introduction of functional genes, in
the form of DNA, to replace mutated genes.This
approach has been used to successfully treat a
number of diseases including X-linked SCID,
ADA-SCID, haemophilia B, lipoproteinlipase
deficiency and Leber's congenital amaurosis with
intensive research on-going for other inherited
diseases. More recently scientists are looking at
using gene technology to repair rather than
replace mutated genes, silence overactive genes
and to also provide our immune cells with the
tools they need to recognise and kill cancer
cells and infections.
What is Gene Therapy
  • Gene therapy is an experimental technique that
    uses genes to treat or prevent disease. In the
    future, this technique may allow doctors to treat
    a disorder by inserting a gene into a patient's
    cells instead of using drugs or surgery.
  • Gene therapy is when DNA is introduced into a
    patient to treat a genetic disease. The new DNA
    usually contains a functioning gene to correct
    the effects of a disease-causing mutation.
  • Gene therapy could be a way to fix a genetic
    problem at its source. By adding a corrected copy
    of a defective gene, gene therapy promises to
    help diseased tissues and organs work properly.

Needs of Gene Therapy
  • To overcome the disease.
  • Targeting the cell of diseased tissues.
  • Genes can be inserted into the cell of patients
    by direct and indirect routes, a inserted the
    genes can integrate into the chromosomes.

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Types of Gene Therapy
Two Types of Gene Therapy
  • Germ line Gene Therapy.
  • Somatic gene Therapy.

Germ Line Gene Therapy
  • Therapeutic genes transferred into the germ
  • It is heritable and passed on to later
  • For safety, ethical and technical reasons, it is
    not being attempted at present.
  • Potential for offering a permanent therapeutic
    effect for all who inherit the target gene.

Somatic Gene Therapy
  • Insertion of therapeutic gene into somatic cells
    like fibroblasts,myoblasts,epithelial cells,
    nervous cells,glial cells etc.
  • This can correct the genetic defect in the
  • However,in somatic cell therapy, Transgene cannot
    be passed on to the siblings etc.
  • Affected only the targeted cells in the patient,
    and it not passed to future generation.

Method Adopted of Gene Therapy
  • Ex-Vivo
  • In-Vivo

Ex Vivo
Ex vivo, where cells are modified outside the
body and then transplanted back in again.
In Vivo
In vivo, which means interior where genes are
changed in cells still in the body.
Gene Therapy
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