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GENE THERAPY

Medical treatment that involves


manipulating the genetic information of
diseased cells to correct a genetic
defect or to provide cells a new function
that allows them to overcome an
alteration.
Medical treatment that involves manipulating the genetic information of diseased
cells to correct a genetic defect or to provide cells a new function that allows
them to overcome an alteration.
1. Replace altered genes.

The mutations can be corrected by gene


surgery, replacing or repairing the
defective gene mutated sequence
2. Inhibit or counteract
harmful effects

It is carried out by targeted inhibition of gene expression. This process is carried blocking
promoters, interfering with the mechanisms of gene expression using RNAs antisense which are
complementary to RNA-m and join them blocking them.
3. Insert new genes.

Suicide genes that destroy their host cell itself or stimulators of


the immune response genes are inserted. You can also insert a
copy of the normal gene to replace the function of the mutant
gene and the correct protein is not produced.
- Inactivate oncogenes.

- Input tumor suppressor genes.

- Introduce suicide genes.

- To introduce genes that increase sensitivity to drugs.

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