Gene therapy aims to change the unhealthy proteins that are produced as a result of disease-causing genes.
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What Is Gene Therapy?
Some diseases are due to a known genetic defect or a gene mutation.
KATERYNA KON / SCIENCE PHOTO LIBRARY / Getty Images
The idea behind gene therapy is to direct the body to produce healthy proteins that do not cause disease.
This therapy involves the delivery of DNA or RNA.
The RNA molecule is an intermediate molecule that is formed in the process of protein production.
Research is ongoing into ways to correct genetic defects that have been associated with certain diseases.
There are different types and methods of gene therapy that are being investigated.
Gene therapy is being evaluated as a potential treatment for both types of mutations.
There are several ways of delivering the corrected DNA or RNA into a persons body.
Most cells in your body are somatic cells.
It is then infused with the correct DNA or RNA sequence.
The viral vector containing the correct gene may be injected into a person for delivery of gene therapy.
Stem Cells
Stem cellsare immature cells that have the potential to develop into different types of cells.
Liposomes
This technique uses a lipid (fat) to deliver the genetic DNA or RNA material.
Why Is Gene Therapy Done?
Some gene mutations direct the body to make disease-causing proteins.
Sometimes the DNA molecule can have a gene inserted into it.
The new cells will then also make healthy products.
This is done by the insertion of a non-mutated gene DNA sequence into a DNA molecule.
Risks of Gene Therapy
There are some known risks of gene therapy.
So far, the most common problem associated with gene therapy is a lack of effectiveness.
However, there are also adverse effects that may occur.
The modified cells will be restored to your body after the treatment.
If this occurs, you may be treated again.
Sometimes immature and healthy cells are transplanted to replace cells that have a disease-causing mutation.
you could talk to your doctor about gene therapy.
Frequently Asked Questions
This therapy is considered safe, but there are risks and side effects.
Cystic Fibrosis Foundation.Gene therapy for cystic fibrosis.
Food and Drug Administration.What is gene therapy?
al.Germline HLA-B evolutionary divergence influences the efficacy of immune checkpoint blockade therapy in gastrointestinal cancer.Genome Med.
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