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Types and Risks of Gene Therapy

Gene therapy can be either somatic or germline. Somatic gene therapy affects only the individual while germline gene therapy affects future generations. There are two main types of somatic gene therapy - in vivo and ex vivo. In vivo gene therapy delivers new genetic material directly to target cells while ex vivo modifies cells outside the body before transplanting them back in. Ex vivo gene therapy can be used to treat loss of function disorders by introducing a functional copy of the missing gene. Gene inhibition therapy aims to introduce a gene that inhibits or interferes with a problematic gene's activity, such as an oncogene promoting cancer growth. However, gene therapy risks include immune reactions, targeting the wrong cells, viral infection, and tumor formation.

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241 views8 pages

Types and Risks of Gene Therapy

Gene therapy can be either somatic or germline. Somatic gene therapy affects only the individual while germline gene therapy affects future generations. There are two main types of somatic gene therapy - in vivo and ex vivo. In vivo gene therapy delivers new genetic material directly to target cells while ex vivo modifies cells outside the body before transplanting them back in. Ex vivo gene therapy can be used to treat loss of function disorders by introducing a functional copy of the missing gene. Gene inhibition therapy aims to introduce a gene that inhibits or interferes with a problematic gene's activity, such as an oncogene promoting cancer growth. However, gene therapy risks include immune reactions, targeting the wrong cells, viral infection, and tumor formation.

Uploaded by

Aqib Khalid
Copyright
© © All Rights Reserved
We take content rights seriously. If you suspect this is your content, claim it here.
Available Formats
Download as PPTX, PDF, TXT or read online on Scribd

Types of Gene Therapy

Introduction
• Somatic gene therapy: Transfer of a section of DNA to any cell of the body that doesn’t
produce sperm or eggs. Effects of gene therapy will not be passed onto the patient’s
children.
• Germ line gene therapy: Transfer of a section of DNA to cells that produce eggs or
sperm. Effects of gene therapy will be passed onto the patient’s children and subsequent
generations
Somatic Gene Therapy Types
• In vivo Gene Therapy
o Delivery of new Genetic material directly to target cells in the body
o Transient expression

o Treatment of cystic fibrosis


o Lungs ,Brain
Ex vivo Gene Therapy
• Cells are modified outside the body and then transplanted back it again
• Long-term expression
• Fibroblast cells, Hematopoietic cells
Ex Vivo Gene Therapy
• This is used to treat diseases caused by a mutation that stops a gene from producing a
functioning product, such as a protein.
• This therapy adds DNA containing a functional version of the lost gene back into the cell.
• The new gene produces a functioning product at sufficient levels to replace the protein
that was originally missing.
• This is only successful if the effects of the disease are reversible or have not resulted in
lasting damage to the body.
• For example, this can be used to treat loss of function disorders such as cystic fibrosis by
introducing a functional copy of the gene to correct the disease
GENE INHIBITION THERAPY
• Suitable for the treatment of infectious diseases, cancer and inherited disease caused by inappropriate
gene activity.
• The aim is to introduce a gene whose product either:
• inhibits the expression of another gene
• interferes with the activity of the product of another gene.
• The basis of this therapy is to eliminate the activity of a gene that encourages the growth of disease-
related cells.
• For example, cancer is sometimes the result of the over-activation of an oncogene (gene which
stimulates cell growth). So, by eliminating the activity of that oncogene through gene inhibition
therapy, it is possible to prevent further cell growth and stop the cancer in its tracks.
Risk of Gene Therapy
• This technique presents the following risks:
• Unwanted immune system reaction. Your body's immune system may see the newly introduced
viruses as intruders and attack them. This may cause inflammation and, in severe cases, organ failure.
• Targeting the wrong cells. Because viruses can affect more than one type of cells, it's possible that
the altered viruses may infect additional cells — not just the targeted cells containing mutated genes.
If this happens, healthy cells may be damaged, causing other illness or diseases, such as cancer.
• Infection caused by the virus. It's possible that once introduced into the body, the viruses may
recover their original ability to cause disease.
• Possibility of causing a tumor. If the new genes get inserted in the wrong spot in your DNA, there is
a chance that the insertion might lead to tumor formation.

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