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Gene Therapy

Gene therapy replaces or adds genes to treat diseases. It works by 1) adding genes to produce missing proteins, 2) inhibiting genes causing disease, or 3) killing specific diseased cells. While promising for cancers, cystic fibrosis, and more, challenges remain in reliably delivering genes and the expense of ongoing treatment.

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Ki xxi
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190 views3 pages

Gene Therapy

Gene therapy replaces or adds genes to treat diseases. It works by 1) adding genes to produce missing proteins, 2) inhibiting genes causing disease, or 3) killing specific diseased cells. While promising for cancers, cystic fibrosis, and more, challenges remain in reliably delivering genes and the expense of ongoing treatment.

Uploaded by

Ki xxi
Copyright
© © All Rights Reserved
We take content rights seriously. If you suspect this is your content, claim it here.
Available Formats
Download as PDF, TXT or read online on Scribd
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What is Gene Therapy?

The primary endorsed gene therapy clinical inquiry within the US took place on 14
September 1990, at the National Establishing of Wellbeing (NIH).

❖ Gene therapy replaces a faulty gene or adds a new gene in an attempt to cure
disease or improve your body's ability to fight disease.
❖ Gene therapy holds promise for treating a wide range of diseases, such as
cancer, cystic fibrosis, heart disease, diabetes, hemophilia and AIDS.

Gene Therapy Technique


1. Gene Augmentation 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.
2. 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.

3. Killing of Specific Cell


● Suitable for diseases such as cancer that can be treated by
destroying certain groups of cells.

● The aim is to insert DNA into a diseased cell that causes that cell to
die.

Advantage of Gene Therapy


❖ The effects of therapy are long lasting and timeliness
❖ Can eradicate diseases and improve the quality of life
❖ Could offer the potential of new discoveries.
❖ Does not just apply to human treatment options.
❖ It allows us to treat the “untreatable” diseases.

Disadvantage of Gene Therapy


❖ Does not have a reliable delivery method.
❖ It is an expensive procedure.
❖ Requires ongoing treatment to be effective.
❖ Might only delay the inevitable.

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