WHAT GENE THERAPY?• A technique used to substitute defective genes in an individual with genes that do work.• These defects cause diseases and illnesses.• The scientific world is trying to understand the way our body reacts without these genes and why our genes sometimes aren’t “on.”
When did it Start?• James Watson and Francis Crick learned about the double helix of DNA in 1953.• Gene therapy evolved in the Recombinant DNA Era, in the 1980s.
What Happens?1. Cut a piece of DNA with restriction enzymes2. Insert the pieces into a plasmid3. Analyze gene4. Define defect or disease5. Make a plan for treatment (example in steps 6 and 7)6. Replace defective cells with “normal” cells7. Insert new cells into patient through vectors
Types of Gene Therapy• Somatic Cells: • Germline Cells:1. Majority of cells in the 1. Cells exist in sperm human body and eggs2. Not passed on to 2. Potential of having future generations negative effect for3. Less controversial and future generations more effective if 3. Potentially most started sooner effective
Somatic Cells• Cells are removed from body before treatment.• Grown in lab, fix gene and insert back into body through blood stream.• Many cells to work with.• Higher risk for older children or young adults.• Used to treat illnesses such as cancer, cystic fibrosis, and muscular dystrophy.
Germline Cells• Correct abnormalities of gene involved in reproduction.• Abnormalities are seen before becoming tissue.• Controversial- “playing God” with unformed tissue.• Most effective during embryonic stage.• Potential negative affects on future generations.
Vector Deliveries 1. EX-VIVO METHOD• Outside living body.• “Normal cells” are inserted in vector.• Blood cells with defective gene are removed from patient.• Patient’s blood is mixed with viruses from vector.• Combination of new and old cells are put back into the patient.• Protein is produced to fight disease.
Vector Deliveries 2. IN-VIVO METHOD• Inside living body.• Does not use patient’s cells.• Vectors are given “new” and “affective” cells.• New cells put into patient’s bloodstream.• New cells bind with targeted diseased cells.• Reverses effect of gene.
BACTOFECTION:Newer method of gene therapy.• Uses bacteria for a direct transfer of the gene to the individual.• Bacterial strains deliver the genes to the plasmid and into the cells.• Bacteria used as “vehicle” to transfer genetic information to the cell.
Positive Aspects• Has “incredible therapeutic potential.”• Potential to find cures to genetic diseases.• Defects can be eliminated and not passed on to future children.• Stops suffering of patient and family.
Negative Aspects• Immune system can destroy the vector.• Very expensive for patient.• “Make the rich, richer, and the poor, poorer.”• “Designer babies” may be made.• Harmful effects.• Against religious beliefs.• “Playing with God and Mother Nature.”
WORKS CITED• Bergeson, Emilie R. "The Ethics of Gene Therapy." The Ethics of Gene Therapy. Web. 15 May 2012. <http://www.ndsu.edu/pubweb/~mcclean/plsc431/students/bergeson.htm>.• Bose, Debopriya. “Gene Therapy Pros and Cons.” Web. 15. 2012. <http://www.buzzle.com/articles/gene-therapy-pros-and-cons.html>• "Gene Therapy Reverses Type 1 Diabetes in Mice, Study Finds." ScienceDaily. ScienceDaily, 06 June 2011. Web. 9 May 2012. <http://www.sciencedaily.com/releases/2011/06/110606092738.htm>.• Nature.com. Nature Publishing Group. Web. 15 May 2012. <http://www.nature.com/gt/journal/v13/n2/full/3302635a.html>.• "Types of Gene Therapy." Â« Walesgenepark.co.uk. Web. 15 May 2012. <http://www.walesgenepark.co.uk/types-of-gene-therapy-2>.