Gene Therapy
Published on Sep 20, 2016
No Description
MORE DECKS TO EXPLORE
PRESENTATION OUTLINE
Gene Therapy
Maleana Khounvongsa
"The process of correcting or modifying DNA codes that cause genetic diseases and disorders."
Two Common Ways To Introduce New Genes Into Defective Cells
Virus
- Carries new or normal genes into target cell
Liposome
- Package a "good" gene in a synthetic lipid envelope and use the envelope to bring gene into cell
Cystic Fibrosis
- Build up of thick mucus that clogs the respiratory and digestive systems. Leads to lung infections and breathing problems.
- 1 in 3,000 babies
- Average lifespan: 35 years
One of the first attempts at human gene therapy
Success
- In 2002, a modified cold virus was used to transfer a normal copy of the gene CFTR to the lining in the nose.
- CFTR regulates the flow of chloride ions into cells and is defective in CF patients. The transferred gene corrected this defect.
CFTR: Cystic Fibrosis Transmembrane Conductance Regulator
Untitled Slide
- Parkinson's disease
- Diabetes
- Cancers
Companies specializing in corrective gene therapy are working on correcting other genes such as:
Photo by stereotyp-0815
Cancer
- In 2006, researchers at the National Cancer Institute used gene therapy to treat advanced melanoma (skin cancer).
- Added genes to white blood cells. When the modified white blood cells were reintroduced into the patient, they better recognized and attacked the melanoma cells.
Researchers hope that the gene therapy techniques they used can be applied to other cancers
Photo by Albaraa Mehdar
