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Gene
Therapy
www.wesrch.com
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.
Types of Gene Therapy
Germ line Gene Therapy.
Somatic gene Therapy.
Two Types of Gene
Therapy
Germ Line Gene Therapy
Therapeutic genes transferred into the
germ cells.
It is heritable and passed on to later
generations.
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
patient.
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
www.wesrch.com
Medical.wesrch.com

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About The Gene Therapy

  • 2. 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.
  • 3. 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.
  • 4. 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.
  • 5.
  • 6. Types of Gene Therapy Germ line Gene Therapy. Somatic gene Therapy. Two Types of Gene Therapy
  • 7. Germ Line Gene Therapy Therapeutic genes transferred into the germ cells. It is heritable and passed on to later generations. 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.
  • 8. 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 patient. 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.
  • 9. Method Adopted of Gene Therapy Ex-Vivo In-Vivo
  • 10. Ex Vivo Ex vivo, where cells are modified outside the body and then transplanted back in again.
  • 11. In Vivo In vivo, which means interior where genes are changed in cells still in the body.