GENE THERAPY

Group 4
@Clark Sevilla
@Edrean Estur Paquita
@Jay D. Cuerdo
@Jess Antonio Buenaventura
@Theodore F. Villariza IV
Gene Therapy
◦ Researchers are testing several approaches to gene therapy, including:
◦ -Replacing a mutated gene that causes disease with a healthy copy of the gene. -Inactivating, or "knocking out," a mutated
gene that is functioning improperly. -Introducing a new gene into the body to help fight a disease.
◦ Gene therapy is designed to introduce genetic material into cells to compensate for abnormal genes or to make
◦ a beneficial protein. If a mutated gene causes a necessary protein to be faulty or missing, gene therapy may be
◦ able to introduce a normal copy of the gene to restore the function of the protein. Researchers must overcome many technical
challenges before gene therapy will be a practical approach to
◦ treating disease. For example, scientists must find better ways to deliver genes and target them to particular
◦ cells. They must also ensure that new genes are precisely controlled by the body. The genetic engineering of humans is not yet
a reality. But, with advancements in gene therapy and cloning, it will be.
 Gene Therapy
◦ A cure
◦ A permanent effect
◦ New gene is inherited
◦ Sense of well being: "I AM NORMAL!"
◦ Hope in living a normal life
Conventional Therapy
◦ Symptomatic/Palliative R
◦ No effect once drug is stopped
◦ Disease is inherited
◦ Constant reminder: "I HAVE A DISEASE..."
◦ Maybe improves quality of life
Approaches of Gene Therapy

◦ 1. Gene Replacement/Correction - Replacing a mutated gene with a healthy copy. Ex.) Severe Combined Immunodeficiency
◦ 2. Gene Silencing/Gene Interference - Inactivating or "knocking out" a mutated gene. Ex.) Sickle Cell Disease
◦ 3. Gene Augmentation/Gene Addition - Introducing a new gene to help fight disease. Ex.) Parkinson's Disease
◦ 4. "Suicide Gene" - Can cause a cell to kill itself through apoptosis - Makes cancer cells more vulnerable and sensitive to
anticancer drugs. Ex.) Solid Tumors Approaches
Different Types of Gene Therapy

1. Germ Line Therapy- altering a gene of an egg or a sperm cell.

2. Somatic Cell Therapy- involves altering the genetic code of a person's somatic cells. Ex Vivo Approach- treating cells
outside the body.
-In Vivo Approach-treatment within the patient's body and uses vectors.
-Vectors for Gene Therapy - Different carrier systems are used for gene delivery.
-NON-viral Vectors (Physical and Chemical)
-Viral Vectors (Biological)
Thank you